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Search for: "Gene therapy" - 250 articles found

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News • Gene Therapy

Innovative gene therapy for hemophilia

Until now, those affected have had to inject the missing coagulation factor proteins themselves several times a week. Gene therapy now offers those affected the prospect of an improvement: the therapeutic agent is administered as a single intravenous injection.

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Article • Knowledge gaps in gender medicine

Covid-19 and sex: higher mortality of male patients

The Covid-19 pandemic has affected scientific research in numerous ways – for example by highlighting knowledge gaps in gender medicine. In many studies differences in morbidity and mortality between women and men surfaced incidentally. While the extent and causes of these differences remain largely unexplored, the preliminary insights confirm the need for further research.

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News • Promising application of "gene scissors"

Using CRISPR-Cas9 to treat anaemia

ETH Zurich molecular biologist Mandy Boontanrart is researching gene therapies that could be used to cure two of the most common types of inherited anaemia: beta thalassaemia and sickle cell anaemia.

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Article • Progress, limitations, and opportunities

Precision oncology: incredible potential, but not a miracle cure

Unanswered questions are hampering clinicians in their efforts to get the best out of a precision medicine approach for their patients. Speaking at the Genomics and Precision Medicine Expo in London at the end of May, cancer educator Dr Elaine Vickers said the benefits of being matched to an investigational drug remain questionable for most people with advanced cancer.

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News • Alveolar rhabdomyosarcoma

Promising new treatment for deadly pediatric tumor

Alveolar rhabdomyosarcoma is a rare pediatric tumor. For more than 40 years there has not been any new development regarding treatment. Research led by Prof. Dr. Anton Henssen at Charité University Berlin has now identified a new therapeutic option, using a drug that is currently under investigation for other types of cancer.

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Article • Antimicrobial resistance development

AMR and climate change: a worrying dual threat to global health

Climate change and antimicrobial resistance (AMR) are forming an alarming alliance: Global warming creates new breeding grounds for resistant bacteria. A serious and very real threat to public health – but not quite the doomsday scenario some might make it out to be, says Prof Sabiha Essack from the University of KwaZulu-Natal in Durban, South Africa.

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News • miR-634 vs. OSCC

Anti-cancer "dream cream" to shrink oral tumors

Modern medicine offers “peel and stick” solutions like nicotine or contraceptive patches to put right on the skin without needing to visit a doctor for an injection or procedure. Now, researchers have found that applying a topical ointment containing anti-tumor factor can increase the effectiveness of cancer treatment.

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News • Fibrodysplasia ossificans progressiva

FOP: Origin of rare disease

A mutation in the gene that causes fibrodysplasia ossificans progressiva (FOP) doesn’t just cause extra bone growth but is tied to a problem in generating new muscle tissue after injury.

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News • Synthetic nucleic acid

New approach could help weak hearts

Heart failure with preserved ejection fraction was previously considered largely untreatable. A research team at the Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC) led by Professor Michael Gotthardt has now succeeded for the first time in improving cardiac function with the help of a synthetic nucleic acid, as the researchers report in the journal Science…

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Article • Mass spec, qPCR and more

Newborn screening: early detection – new diagnostic possibilities

Since its introduction around 60 years ago, the screening of newborn babies for immune, hormone and metabolic disorders has prevented many people from experiencing severe disease progression. The scope of systematic early testing has been significantly enhanced through mass spectrometry (MS). In our interview, Professor Uta Ceglarek, one of the driving forces behind the introduction of MS…

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News • DNA analysis

Should all babies have their genome sequenced at birth?

Genomics England, a government owned company, recently announced a pilot programme of whole genome sequencing to screen for genetic diseases in 200,000 healthy seeming newborns. But should every newborn baby have their whole genome sequenced? Experts debate the issue in The BMJ. Extensive clinical evidence has shown that screening for genetic diseases saves lives, and research has shown that it…

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Article • NGS solution

Using liquid biopsy to detect recurrent breast cancer earlier

An innovative collaboration has been formed in the UK between academic researchers and industry to develop a rapid integrated liquid biopsy platform for early detection of recurrent breast cancer. Breast cancer specialists Professor Charles Coombes, who is Professor of Medical Oncology at Imperial College London (ICL), and Professor Jacqui Shaw, Head of the Department of Genetics and Genome…

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Video • Prevention of triple-negative BRCA

Breast cancer vaccine: clinical trial started

Researchers from Cleveland Clinic have opened a novel study for a vaccine aimed at eventually preventing triple-negative breast cancer, the most aggressive and lethal form of the disease. This phase I trial is designed to determine the maximum tolerated dose of the vaccine in patients with early-stage triple-negative breast cancer and to characterize and optimize the body’s immune response. The…

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News • Cancer research

How to break through a tumor's protective shield

The immune system protects the body from cancer. To protect healthy body cells from its own immune system, they have developed a protective shield: the protein CD47 is a so called "don’t eat me" signal, which tells the immune cells to stand back. Tumor cells exploit this CD47-based protection strategy for evading the immune system, by increasing presentation of CD47 on their cell…

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Sponsored • Tools for the lab

Speeding up diagnostics to detect antibiotic resistance

Infectious disease diagnostics are notoriously slow. The gold standard for laboratory diagnosis of bacterial and fungal infection involves growing the pathogen from a clinical specimen – an overnight event, or even longer. The healthcare focus is on improving the use of antibiotics for better patient outcomes and reducing the environmental pressures that drive antibiotic resistance. To impact…

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News • Amyotrophic lateral sclerosis

New predictive biomarkers for ALS identified

Some blood lipid biomarkers linked to cardiovascular disease risk are also associated with a lower risk of amyotrophic lateral sclerosis (ALS) suggest the findings of a large epidemiology study. ALS is the commonest form of motor neuron disease - a progressive nervous system disease that destroys nerve cells responsible for voluntary movement such as walking and talking.

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News • Understanding oncogenesis

How normal cells turn into liver cancer cells

Researchers at the University of Helsinki could show for the first time that normal human fibroblast cells can be converted to specific cancer cells using only factors that are commonly detected in actual human patients. Previous studies have achieved this only by using powerful viral factors that are not common in human cancers. Since many human cancer types still lack specific diagnostic…

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News • Prevention of toxic DNA lesions

Promising mechanism to stop Huntington's progression

A new mechanism that stops the progression of Huntington’s disease in cells has been identified by scientists at the University of Cambridge and University College London (UCL), as part of their research groups at the UK Dementia Research Institute. Researchers say the breakthrough study, published in Cell Reports, could lead to much needed therapies for the rare genetic disease, which is…

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Article • Precision oncology

Personalized health and genomics: Minimizing collateral damage

A solid diagnosis has always been the first step on any patient’s journey to health. However, diagnostic categories are necessarily oversimplifications. In the last decades, medical professionals and scientists have begun to uncover the true variability in patients’ physiological and biochemical make-up that is the principal cause for individual variations in the way diseases present…

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News • Brain cancer research

Researchers 3D-print entire active tumor

Researchers at Tel Aviv University have 3D-printed a first-of-its-kind glioblastoma tumor that mimics a living cancer malignancy, powering new methods to improve treatment and accelerate the development of new drugs for the most lethal type of brain cancer. Glioblastoma is notoriously fatal as it accounts for the majority of brain tumors and is highly aggressive. The average survival time of…

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Article • Omics in cancer care

Personalizing laboratory medicine

To avoid adverse reactions, personalised laboratory medicine can help to predict a patient’s drug response. Investigations based on DNA and other omics technologies – e.g. genomics, transcriptomics, proteomics, metabolomics – along with microarray technologies, is making a particularly valuable contribution to cancer care, in which personalised approaches are becoming possible through…

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News • Controlling KRAS

New targeted gene therapy could stop lung cancer progression

A newly targeted therapy could help millions of lung cancer patients worldwide keep their cancers from spreading, says an expert at Cleveland Clinic, on the occasion of World Lung Cancer Day. Dr. Khaled Hassan, of the Hematology and Medical Oncology Department at Cleveland Clinic, explains the concept of KRAS targeted therapy – and why the approach should not be mistaken for a cancer cure.

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News • Tool to identify tumour mutations

Machine learning fuels personalised cancer medicine

The Biomedical Genomics laboratory at the Institute for Research in Biomedicine (IRB) Barcelona has developed a computational tool that identifies cancer driver mutations for each tumour type. This and other developments produced by the same lab seek to accelerate cancer research and provide tools to help oncologists choose the best treatment for each patient. The study has been published in the…

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News • Genetic alterations

Same mutation, different cancers: researchers explore connections

Why do alterations of certain genes cause cancer only in specific organs of the human body? Scientists at the German Cancer Consortium (DKTK), the Technical University of Munich (TUM), and the University Medical Center Göttingen have now demonstrated that cells originating from different organs are differentially susceptible to activating mutations in cancer drivers: The same mutation in…

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News • Mechanical inactivation

'Nano traps' to lock up and neutralize viruses

To date, there are no effective antidotes against most virus infections. An interdisciplinary research team at the Technical University of Munich (TUM) has now developed a new approach: they engulf and neutralize viruses with nano-capsules tailored from genetic material using the DNA origami method. The strategy has already been tested against hepatitis and adeno-associated viruses in cell…

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Article • Oncology

Pancreatic cancer - current challenges and future direction

Pancreatic cancer is one of the deadliest cancers in the world, and one of the most difficult to treat. In 2020, an estimated 495,000 individuals worldwide were diagnosed with pancreatic cancer and an estimated 466,000 died, according to statistics from the World Health Organization’s International Agency for Research on Cancer. Most patients with advanced disease die within a year of…

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News • Leading cause for blindness

Breakthrough in research on age-related macular degeneration

Age-related macular degeneration (AMD) is the most common cause of blindness in developed countries affecting seven million in total in Germany, from which 500,000 people are suffering from late stage disease, around half of whom are registered as visually impaired. There are two forms of AMD, ‘wet’ and ‘dry’. There are currently no treatments available for the dry form of the disease…

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News • Unprecedented improvements

Gene therapy 'reprograms' cells to reverse AADC deficiency

A novel method of gene therapy is helping children born with a rare genetic disorder called AADC deficiency that causes severe physical and developmental disabilities. The study, led by researchers at The Ohio State University Wexner Medical Center and The Ohio State University College of Medicine, offers new hope to those living with incurable genetic and neurodegenerative diseases.

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News • Neoadjuvant chemotherapy

Breast cancer: New test predicts therapy success

In a collaboration with the Faculty of Statistics at TU Dortmund and the University Medical Center in Mainz, a research team at the Leibniz Research Centre for Working Environment and Human Factors in Dortmund (IfADo) has developed a test that can be used to predict the success of therapy for breast cancer. Breast cancer is one of the most common tumour diseases worldwide. One in eight women will…

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News • Pediatric oncology cooperation

A royal visit for children with cancer

Queen Máxima of the Netherlands, after whom the Princess Máxima Center for Pediatric Oncology in Utrecht is named, along with several representatives of the center, the Hopp Children’s Cancer Center Heidelberg (KiTZ), University Hospital Heidelberg (UKHD) and the German Cancer Research Center (DKFZ), signed a memorandum on strategic cooperation in the field of pediatric oncology in Europe at…

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News • Transient implant

A pacemaker that dissolves when it's no longer needed

Researchers at Northwestern and George Washington (GW) universities have developed the first-ever transient pacemaker — a wireless, battery-free, fully implantable pacing device that disappears after it’s no longer needed. The thin, flexible, lightweight device could be used in patients who need temporary pacing after cardiac surgery or while waiting for a permanent pacemaker. All components…

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News • „Swarm Learning“

AI with swarm intelligence to analyse medical data

Communities benefit from sharing knowledge and experience among their members. Following a similar principle - called “swarm learning” - an international research team has trained artificial intelligence algorithms to detect blood cancer, lung diseases and Covid-19 in data stored in a decentralized fashion. This approach has advantage over conventional methods since it inherently provides…

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News • Pancreatic ductal adenocarcinoma (PDAC)

Cutting-edge approach to fighting deadly form of pancreatic cancer

By 2030, the most lethal form of pancreatic cancer, pancreatic ductal adenocarcinoma (PDAC), is projected to become the second leading cause of cancer-related deaths in the United States. Not only are therapeutic options limited, but nearly half of PDAC patients who have their tumors removed surgically experience disease recurrence within a year, even with chemotherapy. For more advanced stages,…

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News • Inner ear reseach

Hearing loss: newfound gene gives insights into the cochlea

A gene called GAS2 plays a key role in normal hearing, and its absence causes severe hearing loss, according to a study led by researchers in the Perelman School of Medicine at the University of Pennsylvania. The researchers, whose findings are published online in Developmental Cell, discovered that the protein encoded by GAS2 is crucial for maintaining the structural stiffness of support cells…

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News • Vision for vision

Reversing blindness: Award for cone optogenetics gene therapy

The Foundation Fighting Blindness has granted 600,000 US$ to help Hendrik Scholl as principal investigator define a novel way of reversing blindness. Hendrik Scholl is Director of the Institute of Molecular and Clinical Ophthalmology Basel (IOB), Professor and Chairman of the Department of Ophthalmology, University of Basel, and Head of the University Hospital’s Eye Clinic in Basel, Switzerland.

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News • The stronger sex after all

Why women may be better equipped to fight Covid-19

When it comes to Covid-19, women seem to be the stronger sex, suffering severe disease at about half the rate as men, but the reason for this has been elusive. Now a chance experiment by an ophthalmology researcher at Duke Health has led to an insight: Women have more of a certain type of immune cell that fights infections in mucosal tissue, and these immune cells amass in the lungs, poised to…

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News • Cancer research

'Gene ferry' improves immune therapies

Genetically enhancing a patient's immune cells by adding therapeutic genes to them outside the body is regarded as a promising new treatment approach in oncology. However, the production of these therapeutic cells using viruses is not only expensive but time-consuming. Researchers at the German Cancer Research Center (DKFZ) have developed an innovative non-viral vector that can efficiently…

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News • Reverse genetics system

SARS-CoV-2: An easier and quicker way to analyse mutations

SARS-CoV-2 is the virus responsible for the Covid-19 pandemic. We know that mutations in the genome of SARS-CoV-2 have occurred and spread, but what effect do those mutations have? Current methods for studying mutations in the SARS-CoV-2 genome are very complicated and time-consuming because coronaviruses have large genomes, but now a team from Osaka University and Hokkaido University have…

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News • Biomarker research

"Jumping" genes might protect against AML and other blood cancers

New research has uncovered a surprising role for so-called “jumping” genes that are a source of genetic mutations responsible for a number of human diseases. In the new study from Children’s Medical Center Research Institute at UT Southwestern (CRI), scientists made the unexpected discovery that these DNA sequences, also known as transposons, can protect against certain blood cancers. These…

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News • CRISPR-Cas9

An 'on-off switch' for gene editing

Over the past decade, the CRISPR-Cas9 gene editing system has revolutionized genetic engineering, allowing scientists to make targeted changes to organisms’ DNA. While the system could potentially be useful in treating a variety of diseases, CRISPR-Cas9 editing involves cutting DNA strands, leading to permanent changes to the cell’s genetic material. Now, in a paper published online in Cell,…

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News • Biochemistry

Potential trigger of Crohn’s disease found

Potentially game-changing research led by McMaster University scientists may finally bring relief to millions of people worldwide living with Crohn’s disease. Investigator Brian Coombes said his team identified a strain of adherent-invasive E-coli (AIEC) that is strongly implicated in the condition and is often found in the intestines of people with Crohn’s disease. “If you examine the gut…

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News • Here comes the tooth fairy

New drug shows promise for regenerating lost teeth

A new study by scientists at Kyoto University and the University of Fukui may offer hope for adults who have lost their teeth. The team reports that an antibody for one gene - uterine sensitization associated gene-1 or USAG-1 - can stimulate tooth growth in mice suffering from tooth agenesis, a congenital condition. The paper was published in Science Advances. Although the normal adult mouth has…

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News • Intrahepatic cholangiocarcinoma

A new approach for treating bile duct cancer

Intrahepatic cholangiocarcinoma (ICC) develops within the liver. With one to two cases per 100,000 inhabitants in Germany, ICC is one of the rare diseases overall, but it is the second most common liver cancer. The aggressive bile duct tumour remains clinically inconspicuous for a long time, so that it is often only detected late. Because the tumour also only responds to chemotherapy to a limited…

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News • Breast cancer diagnostics

New system to make AI diagnosis explainable

Researchers at TU Berlin and Charité – Universitätsmedizin Berlin as well as the University of Oslo have developed a new tissue-section analysis system for diagnosing breast cancer based on artificial intelligence (AI). Two further developments make this system unique: For the first time, morphological, molecular and histological data are integrated in a single analysis. Secondly, the system…

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News • A BOLD-100 approach

Novel metallodrug shows promise in tumour treatment

BOLD-100/KP1339 is a ruthenium-based anticancer agent that has been co-developed at the University of Vienna and which has shown promising results in clinical trials in cancer patients. However, the mode of action of this metal compound has not yet been fully elucidated. Researchers from the University of Vienna and the Medical University of Vienna have now been able to demonstrate that BOLD-100…

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Video • Regenerative medicine

Lab-grown ‘mini-bile ducts’ to repair human livers

Scientists have used a technique to grow bile duct organoids – often referred to as ‘mini-organs’ – in the lab and shown that these can be used to repair damaged human livers. This is the first time that the technique has been used on human organs. The research paves the way for cell therapies to treat liver disease – in other words, growing ‘mini-bile ducts’ in the lab as…

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News • Lymphocytosis research

Tracing the onset of leukaemia

B-cell lymphocytosis, a condition in which individuals have increased levels of particular white blood cells, in some cases leads to blood cancer. As an international team of researchers now shows in samples from patients, both diseases exhibit similar epigenetic signatures that are forming very early during the course of the disease. Chronic leukemias often start insidiously. White blood cells…

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News • Mapping the 'family tree' of cancer

Metastasis monitoring: CRISPR tool catches cancer 'in the act'

When cancer is confined to one spot in the body, doctors can often treat it with surgery or other therapies. Much of the mortality associated with cancer, however, is due to its tendency to metastasize, sending out seeds of itself that may take root throughout the body. The exact moment of metastasis is fleeting, lost in the millions of divisions that take place in a tumor. “These events are…

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News • An unexpected and novel target

How our biological clock could save us from prostate cancer

Our biological or circadian clock synchronizes all our bodily processes to the natural rhythms of light and dark. It’s no wonder then that disrupting the clock can wreak havoc on our body. In fact, studies have shown that when circadian rhythms are disturbed through sleep deprivation, jet lag, or shift work, there is an increased incidence of some cancers including prostate cancer, which is the…

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News • "Faces" of the disease

Covid-19: researchers identify at least 5 variants

According to current studies, the Covid-19 disease which is caused by the SARS-CoV-2 coronavirus comprises at least five different variants. These differ in how the immune system responds to the infection. Researchers from the German Center for Neurodegenerative Diseases (DZNE) and the University of Bonn, together with other experts from Germany, Greece and the Netherlands, present these findings…

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Article • Applications of machine learning

Training AI to predict outcomes for cancer patients

Predicting cancer outcome could help with a clinical decision regarding a patient’s treatment. In his keynote speech during the online ‘7th Digital Pathology and AI Congress: Europe’, Johan Lundin, Research Director at the Institute for Molecular Medicine Finland (FIMM) at the University of Helsinki and Professor of Medical Technology at Karolinska Institute, discussed ‘Outcome and…

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News • Intensive care

Protecting lungs from ventilation-induced injury

An unfortunate truth about the use of mechanical ventilation to save the lives of patients in respiratory distress is that the pressure used to inflate the lungs is likely to cause further lung damage. In a new study, scientists identified a molecule that is produced by immune cells during mechanical ventilation to try to decrease inflammation, but isn’t able to completely prevent…

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News • The role of the MLL4 gene

New insights on the Kabuki syndrome

Scientists have known for years that mutations in the MLL4 gene can cause Kabuki syndrome, a rare developmental disorder. How exactly this happens remeained a mystery for long. Now, a new study illuminates new details. The research suggests that MLL4 controls the production of neurons that secrete growth hormone-releasing hormone (GHRH) in a part of the brain called the hypothalamus. Mice without…

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Video • Resistance pulse

New study reveals how melanoma cells survive targeted therapies

In recent years, targeted therapies have cemented their place as some of the most important tools in cancer treatment. These medicines are designed to block specific signals that tumor cells use to grow and spread, while at the same time leaving normal cells unharmed. Targeted therapies can significantly extend patients’ lives, but the benefits are often only temporary. Over time, many cancers…

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News • Childhood cancer

Molecular super-enhancers determine progression of neuroblastomas

Childhood neuroblastomas display extreme differences in the way they develop: they can shrink spontaneously or spread aggressively to healthy tissue. It is molecular super-enhancers that activate the regulatory circuits that steer the tumor down one path or the other. These are the findings of research conducted by scientists from the Hopp Children's Cancer Center Heidelberg (KiTZ), the German…

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News • Brain rejuvenation

Drug reverses age-related mental decline within days

Just a few doses of an experimental drug can reverse age-related declines in memory and mental flexibility in mice, according to a new study by UC San Francisco scientists. The drug, called ISRIB, has already been shown in laboratory studies to restore memory function months after traumatic brain injury (TBI), reverse cognitive impairments in Down Syndrome, prevent noise-related hearing loss,…

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News • Intratumoral B cells

Another side to cancer immunotherapy?

Scientists report on their detailed look at B cells' presence inside tumors. B cells represent the other major arm of the adaptive immune system, besides T cells, and could offer opportunities for new treatments against some kinds of cancers.

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News • Cancer research

New therapeutic approach against leukemia

Leukemia frequently originates from the so-called leukemic stem cell, which resides in a tumor promoting and protecting niche within the bone marrow. Scientists from the Max Planck Institute of Biochemistry in Martinsried, Germany, have found a new way to make these cells vulnerable by specifically dislodging these cells from their niches.

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News • Therapy development

New approach to 'BEAT' COVID-19

The present Coronavirus pandemic with all its effects on society – both health and economic – highlights the urgency of developing new therapies for COVID-19 treatment. At the same time, it demonstrates the necessity to become well prepared for new virus infections we may be facing in the future. To help control the current pandemic and brace for novel pathogens that may cause future…

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Video • List by top clinicians and researchers

Top 10 medical innovations for 2021

An up-and-coming gene therapy for blood disorders. A new class of medications for cystic fibrosis. Increased access to telemedicine. These are some of the innovations that will enhance healing and change healthcare in the coming year, according to a distinguished panel of clinicians and researchers from Cleveland Clinic. In conjunction with the 2020 Medical Innovation Summit, Cleveland Clinic…

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News • For the development of CRISPR/Cas9

Nobel Prize in Chemistry goes to Emmanuelle Charpentier and Jennifer A. Doudna

The Royal Swedish Academy of Sciences has decided to award the Nobel Prize in Chemistry 2020 to Emmanuelle Charpentier from the Max Planck Unit for the Science of Pathogens, Berlin, Germany, and Jennifer A. Doudna from the University of California, Berkeley, USA, “for the development of a method for genome editing”, more commonly known as the 'gene scissors' CRISPR/Cas9.

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News • Distant metastasis-free interval explored

High-risk breast cancers: Mode of detection is linked to patient prognosis

Breast cancers that are detected in the interval between national screening programme mammograms have a worse prognosis than those detected at the time of a screening, even if they have the same biology, according to research presented at the 12th European Breast Cancer Conference. Analysis of results from over eight years’ follow-up of the international MINDACT randomised phase III clinical…

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Article • Overheard at AAIC 2020

Exciting Alzheimer's findings: ’flu vaccines and P-tau217

More than 32,000 people from over 160 countries registered for The Alzheimer’s Association International Conference (AAIC 2020) in July. This largest and most influential international conference on dementia science had to be held virtually this year, when important highlights were aired. The ability to identify individuals at high risk of developing Alzheimer’s Disease (AD), or at early…

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News • Promising approach for eye diseases

Functional human retinas created in a dish

Scientists have generated accurate replications of human retinas in culture that can be used to pinpoint the specific types of cells affected by genetic eye diseases. The culmination of a six-year effort, this achievement will accelerate progress in developing new therapies and was reported in Cell by a team led by Botond Roska at the Institute for Molecular and Clinical Ophthalmology Basel (IOB)…

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News • Coronavirus

“Hotspots” of a corona infection in the human body

An infection with the coronavirus SARS-CoV-2 can affect multiple organs. With this in mind, researchers of the German Center for Neurodegenerative Diseases (DZNE) and Cornell University in the US have investigated cellular factors that could be significant for an infection. To this end, they analysed the activity of 28 specific genes in a wide range of human tissues.

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News • Neuronal disorder

Tracking the onset of ataxias

“Spinocerebellar ataxias” are diseases of the nervous system associated with a loss of motor coordination. A European research alliance headed by the German Center for Neurodegenerative Diseases (DZNE) and the University of Bonn has now registered whether and how symptoms of ataxia developed over the years in around 250 persons at risk, who initially did not show symptoms. This is the first…

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Article • Neuro-oncology

Challenges in brain tumour segmentation

Neuroradiologist Dr Sofie Van Cauter described the challenges to brain tumour image segmentation during the European Society of Medical Imaging Informatics (EuSoMII) annual meeting in Valencia. She also outlined how, when clinically validated, AI could help tackle such problems. The WHO classification of brain tumours has come a long way since first introduced in 1979. The 2016 classification was…

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Article • The difficulty? Unpredictability in the entire process

Immunotherapy for lung cancer patients

Better outcomes, more favourable prognoses – oncologists and their lung cancer patients didn’t dare to dream about it. Finally, there might be hope. The so-called checkpoint inhibitors (immunotherapy drug) have been used successfully, albeit not for every patient. They are a double-edged sword, with risks as well as opportunities, as explained by Professor Cornelia Schäfer-Prokop.

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News • Ophthalmology

Nanoparticles for gene therapy cure eye diseases

Johns Hopkins scientists report the successful use of nanoparticles to deliver gene therapy for blinding eye disease. A uniquely engineered large molecule allows researchers to compact large bundles of therapeutic DNA to be delivered into the cells of the eye.

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News • Light or severe progression

The dangerous dual role of the immune system in COVID-19

Infection with the novel coronavirus SARS-CoV-2 follows a highly variable course: some of those infected do not even notice it, while others become so seriously ill that their lives are placed at risk. Scientists from the Berlin Institute of Health (BIH) and Charité – Universitätsmedizin Berlin and their colleagues from Leipzig and Heidelberg have now discovered that the immune system has a…

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News • Lesion segregation

Explaining the extreme complexity of mutations in tumor genomes

Scientists from the German Cancer Research Center (DKFZ) and the Universities of Cambridge and Edinburgh have been studying the evolution of tumors following chemical damage. They discovered that the DNA lesions caused by the chemical are not eliminated immediately, but are passed on unrepaired over several rounds of cell division. This "lesion segregation" can drive unexpectedly…

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News • New mechanism discovered

UPR: Stress raises cancer cells' chemo resistance

Resistance of cancer cells against therapeutic agents is a major cause of treatment failure, especially in recurrent diseases. An international team around the biochemists Robert Ahrends from the University of Vienna and Jan Medenbach from the University of Regensburg identified a novel mechanism of chemoresistance which has now been published in "Nature Communications". It is driven by…

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News • Reversing incurable cause for blindness

Macular degeneration: Gene therapy restores vision

Macular degeneration is one of the major reasons for visual impairment, round the globe, close to 200 million people are affected. It damages the photoreceptors in the retina, which lose their sensitivity to light. This can lead to impaired vision or even complete blindness. Scientists at the Institute of Molecular and Clinical Ophthalmology Basel (IOB) together with colleagues from the German…

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News • Cancer research

Lymph node analysis to hunt down metastases

What makes tumor cells turn murderous? The Fraunhofer Institute for Toxicology and Experimental Medicine ITEM is investigating the mechanisms of metastasis formation – and searching for approaches for new treatments in the fight against cancer. Among other things, the research team at Fraunhofer ITEM has developed a method that enables them to analyze entire lymph nodes.

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News • New gene therapy approach

Tumor-tropic technology for targeted cancer therapy

Toshiba Corporation and a team led by Professor Yozo Nakazawa at the Department of Pediatrics, Shinshu University, have together developed a “tumor-tropic liposome technology” for gene therapy. The technology uses unique, nano-sized biodegradable liposomes developed by Toshiba to accurately and efficiently deliver therapeutic genes to targeted cancer cells, and achieves safer gene delivery…

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News • HNSCC diagnostics

Head and neck cancer: Novel prognostic biomarker could double survival

A recent study conducted by the Faculty of Medicine at The Chinese University of Hong Kong (CU Medicine) discovered a novel genetic biomarker which can predict the survival of head and neck cancer patients. There are over 0.7 million new head and neck squamous cell carcinoma (HNSCC) cases globally each year. However, currently there is no clinical implementation of any genetic biomarker to…

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Article • A more integrative approach to digital pathology

imCMS: The door to simple, cheap, reliable bio-stratification

Bringing molecular and digital pathology closer together through a more integrative approach can lead to clear advantages for diagnostic and research workflows. During the recent Digital Pathology and AI Congress (London), Professor Viktor Koelzer explored the benefits and paid particular attention to colorectal cancer (CRC).

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News • A look at oligodendrocytes

Parkinson’s disease may start in the gut

Researchers at Karolinska Institutet and the University of North Carolina have mapped out the cell types behind various brain disorders. The findings are published in Nature Genetics and offer a roadmap for the development of new therapies to target neurological and psychiatric disorders.

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Article • Tools for practitioners

Computational pathology: Heading for personalised medicine

Computational pathology has increased applications for diagnosis, prediction of prognosis and therapy response, facilitating the movement of healthcare towards personalised medicine. Coupled with deep learning, such tools are ever more efficient and robust within research and clinical settings. The growing role of computational pathology was highlighted by Professor Andrew Janowczyk at the…

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News • Amyloid beta regulation

CRISPR helps identify potential Alzheimer's-related protein

Experts at the University of Tokyo have identified a new protein in the pathway that leads to Alzheimer’s disease. Researchers used the “molecular scissors” of CRISPR/Cas9 to search for new genes related to the neurodegenerative disease. The exact causes of Alzheimer’s disease remain unknown, but one of the most well- supported theories focuses on a protein called amyloid beta.…

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News • Cause for colorectal carcinoma

Loss of protein can drive intestinal cancer

An international team of researchers from the University of Zurich, the University Hospital Zurich, Heidelberg and Glasgow has identified a novel function for the cell death regulating protein MCL1: It is essential in protecting the intestine against cancer development – independent of bacterial-driven inflammation. These findings have implications for the use of MCL1 inhibitors, currently…

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News • Altered chromosomes

Breast cancer: targeted therapy can lead to treatment resistance

If chromosomes are unevenly distributed or otherwise altered during cell division, this normally damages the daughter cells and impairs their viability. Not in cancer cells, however, in which chromosome instability can actually confer a growth advantage under certain circumstances. Moreover, as scientists from the German Cancer Research Center have now demonstrated in mice, changes in the…

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News • Releasing the brakes on Duchenne muscular dystrophy

Potential new methods for DMD therapies

Researchers identified a group of small molecules that may open the door to developing new therapies for Duchenne muscular dystrophy (DMD), an as-yet-uncured disease that results in devastating muscle weakening and loss. The molecules tested by the team from the Perelman School of Medicine at the University of Pennsylvania eased repression of a specific gene, utrophin, in mouse muscle cells,…

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Article • On the way to diagnostic mainstream

Liquid biopsy to advance cancer diagnosis

Liquid biopsy offers a new dimension to detection and stratification of cancer – yet the technique also faces hurdles in becoming a mainstream diagnostic approach for more personalised treatments. A critical challenge lies in identifying the extremely low concentrations of the bio-analytes of CTC (circulating tumour cells), ctDNA (circulating tumour DNA) and exosomes in the blood.

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News • Promising metabolite

Gut flora could hold the key for new diabetes treatments

An organic compound produced by the gut flora – the metabolite 4-Cresol – is considered to have protective effects against both type 1 and 2 diabetes, notably by stimulating the growth of the insulin-producing pancreatic beta cells. This is according to a new study led by Inserm researcher Dominique Gauguier at the Environmental Toxicology, Therapeutic Targets, Cell Signaling and Biomarkers…

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News • Blood coagulation

Treating hemophilia with gene therapy

Within the framework of an international study, Lund University and Skåne University Hospital have started treating patients with hemophilia with gene therapy, something that began in January this year. The hope is that the new treatment will significantly simplify everyday life for those with severe hemophilia. Hemophilia is a genetic disease where the body does not produce one of the clotting…

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News • Sex-specific genes

Heart valve defect: female and male hearts react differently

When the heart valve between the aorta and the left ventricle is narrowed, i.e. aortic valve stenosis is present, different genes are active in men than in women. Scientists of the German Centre for Cardiovascular Research (DZHK) at Charité – Universitätsmedizin Berlin have discovered this for the first time. Future research can be planned more precisely according to these results and could…

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News • Gastric squamous-columnar junction cancer

The role of stem cells in deadly gastric SCJ cancer

A study led by scientists from Cornell University provides important new insights into a common and deadly type of gastric cancer. Incidence of this cancer, called gastric squamous-columnar junction (SCJ) cancer, also known as gastroesophageal cancer, rose 2.5 times in the United States between the 1970s and 2000s, while cases of all gastric cancers have decreased by more than 80% since the…

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News • Detailed map of immune cells

The Human Blood Atlas: a mighty new tool to fight deadly diseases

A first-ever map of the human body’s immune cells has been created by scientists at SciLifeLab, providing medical research with a detailed description of the proteins in human blood. The open-access database offers medical researchers an unprecedented resource in the search for treatments for diseases. Published in the journal Science, the Blood Atlas resource is the latest database to be…

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Article • The future has begun

Cancer care 2035: multi-disciplinarity is key

An enthralling insight into the care that could be offered to cancer patients of the future was presented by cancer imaging expert Professor Regina Beets-Tan during her a keynote presentation at the recent British Institute of Radiology congress. In the session ‘Oncologic imaging: Future perspectives’, the professor outlined what a Multi-Disciplinary Team (MDT) of the future – a team in…

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News • Direct-from-blood diagnostic

T2Resistance Panel receives CE mark

T2 Biosystems, Inc., a leader in the development and commercialization of medical diagnostic products, and CARB-X, a global non-profit partnership dedicated to accelerating R&D innovation to address the rising global threat of drug-resistant bacteria, announced the granting of a CE mark to the T2Resistance Panel. With the CE mark, T2 Biosystems has met the requirements of the In-Vitro…

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News • Rapid emergence of antibiotic resistance

How P. aeruginosa becomes resistant during CF treatment

Antibiotic-resistant pathogens pose one of the greatest threats to public health worldwide. In the near future, harmless bacterial infections may no longer be treatable and may again become the most common non-natural cause of death. At the same time, the available repertoire of antibacterial agents is becoming increasingly smaller as resistance rates rise.

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Interview • Chronic inflammations

GATA-3: 'Switching off' allergies and asthma

Chronic inflammatory diseases, such as allergies and asthma, are not only an acute problem but also a major research and prevention challenge. We spoke with Professor Harald Renz, Director of the Institute for Laboratory Medicine at the University Hospital Gießen/Marburg, Germany, and discussed the major reason for increases in the number of these widespread diseases.

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Article • Robotic assistance for middle and inner ear procedures

Cochlear implant microsurgery progresses

Unlike other surgical specialties, ear nose and throat (ENT) has been poorly served by the introduction of robotic platforms to enhance procedures. Since the da Vinci system first gained FDA approval in 2000, robot-assisted surgery has become commonplace in many specialties, including neurology, urology, etc. with numerous other general surgical applications. However, existing systems including…

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News • Promising preclinical study results

Epilepsy: Gene therapy shows long-term suppression of seizures

Teams of researchers from Charité – Universitätsmedizin Berlin and the Medical University of Innsbruck have developed a new therapeutic concept for the treatment of temporal lobe epilepsy. It represents a gene therapy capable of suppressing seizures at their site of origin on demand. Having been shown to be effective in an animal model, the new method will now be optimized for clinical use.…

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News • Iron dependent cell death

Ferroptosis could be key for new anticancer approach

A team of researchers lead by Helmholtz Zentrum München and the University of Würzburg identified an enzyme as a novel and strong inhibitor of ferroptosis, the iron dependent form of cell death: ferroptosis suppressor protein-1, short FSP1. This protein is expressed in a variety of cancer cell lines and therefore represents an attractive drug target for cancer treatment.

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News • Lumacaftor-ivacaftor

Cystic fibrosis patients benefit from drug combination, but...

In adolescent and adult patients with cystic fibrosis (CF) taking lumacaftor-ivacaftor (Orkambi), the combination drug appears to improve lung function and body weight and reduce the need for intravenous antibiotic treatment, according to a French study published online in the American Thoracic Society’s American Journal of Respiratory and Critical Care Medicine. However, the treatment also…

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News • Difficult decisions

5 things women under 40 should know about breast cancer

Breast cancer is rare for women under 40. So, a breast cancer diagnosis can be shocking news for a young woman to hear. “Breast cancer in young women can have its own risk factors and traits, and young women have their own considerations when deciding on a treatment,” says Nadine Tung, MD, head of breast medical oncology and cancer genetics at Beth Israel Deaconess Medical Center (BIDMC).…

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News • New transfection approach

Immunotherapy: The 'FedEx and UPS equivalent of DNA delivery'

Immunotherapy is a promising cancer treatment that uses genetically modified immune cells to fight cancer. It can be used as a primary treatment or in combination with other treatments such as radiation and chemotherapy to slow down or stop the growth of cancer cells and prevent them from spreading to other parts of the body. Chimeric Antigen Receptor (CAR)-T cell therapy, for instance, is a…

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News • Watson on the case

Personalised cancer care through AI

The Geneva University Hospitals (HUG) is the first European university hospital to utilize IBM’s artificial intelligence (AI) technology to help uncover therapeutic options for cancer patients. HUG will use the IBM Watson Health’s precision oncology offering, Watson for Genomics, an AI tool that enables oncologists to provide patients with more personalized, evidence-based cancer care. Using…

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Article • Cancer of unknown primary

CUP: in search for the smoking gun

Cancer of unknown primary (CUP) can send radiologists on a frustrating scavenger hunt: metastases were detected but the primary cancer is nowhere to be seen. Professor Alwin Krämer, Head of the Clinical Cooperation Unit Molecular Haematology/Oncology at University Hospital Heidelberg and the German Cancer Research Center, explains strategies for dealing with CUP.

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Article • After myocardial infarction

Patching up a damaged heart

Scientists in the UK have developed tiny patches of engineered heart tissue that have the potential to be implanted to help people recover from a heart attack. Measuring approximately 3cm x 2cm, the patches contain up to 50 million human-induced pluripotent stem cell derived cardiomyocytes (hiPSC-CM). Yet, these are programmed to turn into working heart muscle that can beat and gradually be…

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News • Stem cell regeneration

Drug accelerates recovery after chemo, radiation

A drug developed by US physician-scientists and chemists speeds up the regeneration of mouse and human blood stem cells after exposure to radiation. If the results can be replicated in humans, the compound could help people recover quicker from chemotherapy, radiation and bone marrow transplants. The study, published in Nature Communications, also sheds light on the basic biology behind blood…

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News • Gene editing

Taking CRISPR one step further

Researchers at ETH Zurich have refined the famous CRISPR-Cas method. Now, for the very first time, it is possible to modify dozens, if not hundreds, of genes in a cell simultaneously. The biotechnological method CRISPR-Cas offers a relatively quick and easy way to manipulate single genes in cells, meaning they can be precisely deleted, replaced or modified. Furthermore, in recent years,…

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News • Personalised medicine

Why digital twins could be the ideal therapy testbed

Advanced computer models of diseases can be used to improve diagnosis and treatment. The goal is to develop the models to “digital twins” of individual patients. Those twins may help to computationally identify and try the best medication, before actually treating a patient. The models are the result of an international study, published in the open access journal Genome Medicine.

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News • Oncology

Response to gene-targeted drugs depends on cancer type

Cancers with the same genetic weaknesses respond differently to targeted drugs depending on the tumour type of the patient, new research reveals. The study is set to prompt changes in thinking around precision medicine—because it shows that the genetics of a patient's cancer may not always be enough to tell whether it will respond to a treatment. The researchers are already starting to design…

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Video • Digital twin

Collaboration of the future: and AI makes three

In view of the advent of personalised medicine and holistic therapy many experts predict the end of healthcare as we know it. However, in many places it is ‘healthcare business as usual’. In our interview, Dr Christoph Zindel, President Diagnostic Imaging at Siemens Healthineers, explains where he sees radiology bridging the gap between symptom-centred treatment today and the systemic…

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Article • Prostate cancer data

Mapping the inflammatory landscape

Image analysis of prostate cancer is a challenging area for clinicians. The disease shows a low mutation burden compared to melanoma and stomach cancer, for example, making morpho-molecular correlation more difficult, and there is often very low inflammation. With the role of tumour infiltrating lymphocytes in prostate cancer currently unclear – and with the advent of new approaches to prostate…

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News • Macular Degeneration

Implanted drug ‘reservoir’ reduces injections

In a clinical trial of 220 people with “wet” age-related macular degeneration, Johns Hopkins Medicine researchers, collaborators from many sites across the country, and Genentech in South San Francisco have added to evidence that using a new implant technology that continuously delivers medication into the eyes is safe and effective in helping maintain vision and reduces the need for…

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News • Oncology

Killing the unkillable cancer cells

We all know someone affected by the battle against cancer. And we know that treatments can be quite efficient at shrinking the tumor but too often, they can’t kill all the cells, and so it may come back. With some aggressive types of cancer, the problem is so great that there is very little that can be done for the patients.

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News • New discovery

Cancer drugs promote stem cell properties of colorectal cancer

Scientists from the German Cancer Research Center (Deutsches Krebsforschungszentrum, DKFZ) and the Mannheim University Medical Center have now discovered that a certain group of cancer drugs (MEK Inhibitors) activates the cancer-promoting Wnt signalling pathway in colorectal cancer cells. This can lead to the accumulation of tumor cells with stem cell characteristics that are resistant to many…

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Article • Flow cytometry

Living cells – the essential biomarker

The team of the Heinz Nixdorf Chair of Biomedical Electronics at the Technical University of Munich focuses on innovative diagnostic tools to accelerate the development towards personalized medicine. Therapies tailored to the individual patient – this is the future not only of oncology but of many medical disciplines. “At this point, however,” concedes Professor Dr Oliver Hayden,…

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News • Induced pluripotent stem (iPS)

Researchers find way to generate stem cells more efficiently

Induced pluripotent stem (iPS) cells are among the most important tools in modern biomedical research, leading to new and promising possibilities in precision medicine. To create them requires transforming a cell of one type, such as skin, into something of a blank slate, so it has the potential to become virtually any other kind of cell in the body, useful for regenerative therapies for…

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News • ctDNA vs. HGSOC

Taking 'molecular snapshots' of ovarian cancer

High-grade serous ovarian cancer (HGSOC) is the most common and aggressive subtype of ovarian cancer. The HGSOC tumors consist of several heterogeneous cell populations with a large number of mutations. This genetic variability makes it difficult to find drugs that would kill all the cancer cells, and to which the cells would not become resistant during treatment. Over half of the patients…

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News • Targeted treatment

Cancer: Blood sample to help select the right early phase clinical trials

Scientists could help match cancer patients with no other treatment options to clinical trials with experimental medicines, by analysing the genetic faults in a sample of their blood. The researchers, funded by Cancer Research UK, The Christie Charity, AstraZeneca and the NIHR Manchester Biomedical Research Centre (BRC), demonstrated in their feasibility study that a blood test can be carried out…

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Article • Differentiate and select

Myths and truths about antibiotics, antiseptics and vaccination

Sixty-two percent of Germans fear antibiotic resistance, according to a survey recently conducted by the German Federal Institute for Risk Assessment. ‘Patients colonised by multi-resistant pathogens are particularly scared. But many of these fears are rooted in misunderstandings,’ explained Professor Mathias Pletz at the Congress for Infectious Diseases and Tropical Medicine (KIT).

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News • CMT, Alzheimer's, Parkinson's

Study links key protein to Charcot-Marie-Tooth and other nerve diseases

A new study provides critical insight into a little-known, yet relatively common, inherited neurological condition called Charcot-Marie-Tooth disease (CMT). The findings point to a pathway to possible treatments for this disease and better understanding of other neurodegenerative disorders, including Alzheimer's disease, that affect millions. The study focused on two related proteins, MFN2 and…

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News • Rare bone cancer

Targeted approach to therapy for chordomas

Chordomas are rare bone tumors for which only few options of treatment exist. Scientists and doctors from the National Center for Tumor Diseases (NCT), the German Cancer Research Center (DKFZ), and Heidelberg University Hospital (UKHD) have discovered a particular genetic trait of chordomas in advanced stages after conducting gene analysis. Their findings, published in the journal Nature…

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News • Tumor-associated macrophages

Immune cells key to predicting cancer outcomes

Scientists have identified key changes in immune cells within cancerous tumours that could help improve the development of treatments. The study, which has been published in the journal Cancer Cell, also found a set of genes that are expressed at high levels in breast cancer tumours and linked to more aggressive cancer types. Researchers say the discoveries offer clues to diagnosis and predicting…

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News • Rapid diagnostics

T2 Biosystems hosts integrated symposium at ECCMID 2019

T2 Biosystems, maker of rapid diagnostic technology to aid in the detection of blood stream infections to prevent sepsis, will host an integrated symposium titled “Rapid Diagnostics Direct from Whole Blood: A Solution for Fast and Appropriate Antimicrobial Therapy,” at the European Congress of Clinical Microbiology & Infectious Diseases (ECCMID) in Amsterdam, Monday, April 15, 16:00-18:00…

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News • Virology

Trapping viruses inside a cell: harmful or helpful?

Viruses are often used as vehicles for delivery in gene therapy because they’re engineered not to damage the cell once they get there, but neglecting to consider how the virus will exit the cell could have consequences. Some viruses use a molecule called heparan sulfate to help them attach to cells. The molecule, found in many different kinds of cells (including those from animal tissue), could…

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