
News • mRNA-based strategy
Cystic fibrosis: new study identifies gene therapy
Now, a new study identified a gene therapy for individuals with cystic fibrosis (CF) caused by a specific mutation and for whom there was no treatment available until now.

Now, a new study identified a gene therapy for individuals with cystic fibrosis (CF) caused by a specific mutation and for whom there was no treatment available until now.

Not all cancer mutations are equal: new research shows that a single mutation hotspot can generate a rich diversity of tumour behaviours. This could lead to more personalised cancer treatments.

Gene-editing technologies show great promise for medical treatments and research, with the potential to cure thousands of genetic diseases. At the 2025 World Medical Innovation Forum in Boston, leading experts explored the possibilities and challenges of these rapidly advancing tools. The case of Baby KJ Muldoon – an infant treated with a personalised CRISPR therapy developed in just seven…

A research team at the University of Geneva has identified two new proteins that regulate gene expression, a discovery that could pave the way for new cancer and brain disorder treatments.

A new way to inhibit the proliferation of Sars-CoV-2 opens up new perspectives in the fight against this coronavirus and other viral diseases that still have no medical treatment.

Researchers discovered a gene that might be a key factor in the progression of Huntington’s disease in organoids. The gene may contribute to brain abnormalities much earlier than previously thought.

This February, the Norwegian Institute of Public Health officially launched the ‘Joint Action on cancer and other non-communicable disease prevention project‘ (JA PreventNCD). In the four-year-initiative, 22 EU member states plus Iceland, Norway and Ukraine have joined forces to combat the increasing burden of non-communicable diseases (NCDs) across national borders.

It's an important milestone for gene editing technology: a novel treatment for sickle cell disease utilizing CRISPR-Cas9, has now received FDA approval – a first for this type of therapy.

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.

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.

At this year's Medica tradefair, laboratory medicine, medical technology and life sciences give new impulses to the entire healthcare market - especially in times of the corona pandemic. An overview.

With the rise of syndrome-style infections, co-infections and the current antimicrobial resistance challenges, the need for multiplexed diagnostics is now more important than ever.

Researchers identify critical spots on the genome where gene editing could cause an unwanted response, and they provide recommendations for safer approaches.

A new approach makes use of natural DNA repair machinery and provides a foundation for novel gene therapy strategies with the potential to cure a large spectrum of genetic diseases.

An ECRC research team has introduced CRISPR-Cas9 into human muscle stem cells for the first time using mRNA, thus discovering a method suitable for therapeutic applications.

Considerable advances in point-of-care testing (POCT) devices are emerging from lab-on-a-chip platforms, innovations in smartphone-based technology and wearable technology. Cloud-based deep learning systems herald a future revolution.

A new paper co-written by a University of Illinois Urbana-Champaign scholar who studies the legal and ethical implications of advanced biotechnologies outlines an unexplored tool to regulate the medically and ethically dubious practice of heritable human-genome editing: patent law.

A research team led by scientists in the labs of Jennifer Doudna, David Savage and Patrick Hsu at the University of California, Berkeley, is aiming to develop a rapid Covid-19 diagnostic test that is much faster and easier to deploy than qRT-PCR. It has now combined two different types of CRISPR enzymes to create an assay that can detect small amounts of viral RNA in less than an hour. Doudna…

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,…

Scientific and public health experts have been raising the alarm for decades, imploring public officials to prepare for the inevitability of a viral pandemic. Infectious epidemics seemingly as benign as "the flu" and as deadly as the Ebola virus provided ample warning, yet government officials seemed caught off guard and ill prepared for dealing with Covid-19.

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…

Scientists at UC Berkeley and Gladstone Institutes have developed a new CRISPR-based COVID-19 diagnostic test that, with the help of a smartphone camera, can provide a positive or negative result in 15 to 30 minutes.

Researchers at Tel Aviv University (TAU) have demonstrated that the CRISPR/Cas9 system is very effective in treating metastatic cancers, a significant step on the way to finding a cure for cancer.

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.

A new way to deliver therapeutic proteins inside the body uses an acoustically sensitive carrier to encapsulate the proteins and ultrasound to image and guide the package to the exact location required, according to Penn State researchers.

Research into the novel Wuhan seafood market pneumonia virus, the deadly "coronavirus" that has forced the Chinese government to quarantine more than 50 million people in the country's dense industrial heartland, will be facilitated by the UC Santa Cruz Genomics Institute. The Genomics Institute's Genome Browser team has posted the complete biomolecular code of the virus for researchers…

A team from the Broad Institute of MIT and Harvard has developed a new CRISPR genome-editing approach by combining two of the most important proteins in molecular biology – CRISPR-Cas9 and a reverse transcriptase – into a single machine. The system, called “prime editing,” is capable of directly editing human cells in a precise, efficient, and highly versatile fashion.

A genetic mutation that a Chinese scientist attempted to create in twin babies born last year, ostensibly to help them fend off HIV infection, is also associated with a 21% increase in mortality in later life, according to an analysis by scientists from UC Berkeley. The researchers scanned more than 400,000 genomes and associated health records contained in a British database, UK Biobank, and…

A proof-of-principle study shows that gold nanoparticles loaded with CRISPR and other gene-editing tools safely and effectively edited blood stem cells.

Tinnitus, more commonly known as ringing in the ears, is a serious audiological and neurological condition affecting nearly 50 million Americans. Noise and music exposure are the predominant environmental risk factors for tinnitus. There is no known cure, and there are no FDA-approved medications developed specifically to treat it. The fact is, tinnitus is very common but not well understood. A…

In the last six years, a tool called CRISPR-Cas9 has transformed genetic research, allowing scientists to snip and edit DNA strands at precise locations like a pair of tiny scissors. But sometimes, it takes more than scissors to do the job. Now, a collaborative international team has unveiled a new CRISPR-based tool that acts more like a shredder, able to wipe out long stretches of DNA in human…

For some cancers, initial treatment with chemotherapy brings positive, but only temporary, results: tumors shrink, but then rebound as the cancer becomes drug-resistant. This pattern of remission-resistance-relapse is particularly true for pancreatic cancer, an aggressive disease in which early success is often countered by eventual disease progression. To wit: The one-year relative survival rate…

How can we detect the first signs of disease as early as possible? Could closer investigation at the cellular level help to quickly prevent disease progression through appropriate treatment? The European Union is now investing a million euros over a one-year period to devise the plan for a fundamentally new approach to understanding the constant changes within cells and their relationships to one…

Scientists of the Helmholtz Centre for Infection Research (HZI) have now dissected the precise molecular mechanisms of WAVE complex activation, occurring in a process involving separable, independent steps.

The CRISPR genome editing technique promises to be a "transformative leap" in genetic engineering and therapy, affecting almost every area of medicine. That includes plastic surgery, with potential advances ranging from prevention of craniofacial malformations, to therapeutic skin grafts, to new types of rejection-free transplants, according to a paper in the November issue of Plastic…

Cancer cells often have mutations in their DNA that can give scientists clues about how the cancer started or which treatment may be most effective. Finding these mutations can be difficult, but a new method may offer more complete, comprehensive results. A team of researchers has developed a new framework that can combine three existing methods of finding these large mutations - or structural…

Austrian researchers have accomplished an astounding feat: They created organoids that mimic the onset of brain cancer. This method not only sheds light on the complex biology of human brain tumors but could also pave the way for new medical applications.

A scientist has discovered a gene-editing technology that could efficiently and accurately correct the genetic defects that underlie certain diseases, positioning the new tool as the basis for the next generation of genetic therapies.

CRISPR has recently become synonymous with precise genetic editing. However, the mighty tool has been known to fail from time to time. Now, researchers have found out why.

Using a new computational strategy, researchers have identified 29 genetic changes that can contribute to an aggressive childhood cancer. Their work helps to explain “the engine” driving formation of rhabdomyosarcoma and suggests potential treatments.

A powerful genome editing tool can be deployed as an ace DNA detective, able to sniff out DNA snippets that signal viral infections, cancer, or even defective genes. This genetic detective, developed in the laboratory of Howard Hughes Medical Institute (HHMI) Investigator Jennifer Doudna at the University of California, Berkeley, uses the genome-slicing system known as CRISPR. By combining the…

UCLA researchers have developed the first map of gene regulation in human neurogenesis, the process by which neural stem cells turn into brain cells and the cerebral cortex expands in size. The scientists identified factors that govern the growth of our brains and, in some cases, set the stage for several brain disorders that appear later in life. The human brain differs from that of mice and…

Using molecular scissors wrapped in a greasy delivery package, researchers have disrupted a gene variant that leads to deafness in mice. A single treatment involving injection of a genome editing cocktail prevented progressive hearing loss in young animals that would have otherwise gone deaf, Howard Hughes Medical Institute (HHMI) Investigator David Liu and colleagues report in the journal…

A research team in the United States has developed a revolutionary technique that has encoded an image and short film in living cells. Scientists at the Wyss Institute for Biologically Inspired Engineering and Harvard Medical School (HMS) used CRISPR gene editing to encode the image and film in DNA, using this as a medium to store information and produce a code that relates to the individual…

After this year's successful 1st congress about Emerging Technologies in Medicine (ETIM) in Essen, the organizing committee sets the stage for the second edition in February 16/17, 2018. This year, the event is dedicated to the role of artificial intelligence and robotics in medicine.

Modifying a DNA sequence to alter a cell to cure a disorder, prevent a genetic disease being passed on to next generations, or to use genetic modification as a way to diagnose a virus: yes, these tasks are possible. The recently discovered CRISPR technique can do it all.

Genomic Health announced that the 15th St. Gallen International Breast Cancer Conference Expert Panel endorsed the use of genomic tests in early-stage breast cancer and recognised the Oncotype DX Breast Recurrence Score test for its prognostic ability as well as its value in guiding treatment decisions on adjuvant chemotherapy for patients with early-stage, endocrine sensitive, invasive breast…

Scientists on the Florida campus of The Scripps Research Institute (TSRI) have improved a state-of-the-art gene-editing technology to advance the system’s ability to target, cut and paste genes within human and animal cells—and broadening the ways the CRISPR-Cpf1 editing system may be used to study and fight human diseases.

According to Darwin, humans will one day become extinct. Some don’t think we need to accept this fate because gene editing may allow us to use our brains to take over the evolutionary story.

When a Lake Malawi cichlid loses a tooth, a new one drops neatly into place as a replacement. Why can't humans similarly regrow teeth lost to injury or disease?

It may come as a bit of a surprise to learn that bacteria have an immune system – in their case to fight off invasive viruses called phages. And like any immune system – from single-celled to human – the first challenge of the bacterial immune system is to detect the difference between “foreign” and “self.” This is far from simple, as viruses, bacteria and all other living things…

Scientists from the Hubrecht Institute and the University Medical Center Utrecht (UMC Utrecht) have developed a cell culture model of human colon cancer progression. This model mimics the situation in patients more closely than any other colon cancer model so far. It enables researchers to study processes involved in colon cancer development and find new cancer drugs.

Researchers at the Hubrecht Institute and the University Medical Center (UMC) Utrecht successfully developed a culturing system for human liver stem cells as well as stem cells from pancreatic cancer. They describe the development of these culturing systems in two articles in this week’s edition of Cell magazine.

99.9% of our DNA is shared with all other humans. Only about 0.1% of our DNA makes us individual