
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.

Why does the same genetic mutation cause FCDII in some patients but not in others? Researchers developed organoids to model a brain malformation responsible for drug-resistant epilepsy in children.

Researchers have now investigated whether a person’s genetic predisposition to different diabetes subgroups can help assess the risk of developing coronary artery disease.

Type 2 diabetes and high blood pressure share a genetic link, new research from the UK and France shows. People with one condition are more likely to develop the other.

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.

Why does Huntington’s disease begin at very different ages? Using advanced AI techniques, neuroscientists from the University of Barcelona found a way to better answer this question.

DNA analysis of colorectal polyps provides important additional information on the development of these polyps and colorectal cancer, research finds. This leads to better diagnostics and treatment.

Scientists have overturned a long-held belief in genetics: that inheriting two harmful variants in the same gene always worsens disease. Instead, this can actually restore normal protein function.

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…

Breast cancer screening for women under 50 isn't cost-effective – yet 1 in 5 diagnoses occurs in this age group. A new study suggests a personalised approach that could change this equation.

New hope for patients with neurological diseases: A combination of focused ultrasound and gene therapy enables targeted, nonsurgical control of seizure-relevant brain regions.

With Taipei City Government's support, six Taipei companies will showcase innovative biotech and medical device solutions at MEDICA 2025 (Nov 17–20, Düsseldorf), advancing healthcare with speed and precision.

In the NICU, a few hours can mean the difference between unnecessary procedures and targeted, life-saving treatment. A new speed record for WGS opens new diagnostic possibilities.

Breast cancer claims around 670.000 lives each year. Now, researchers propose that whole genome sequencing (WGS) could help many patients find better treatments or match them with clinical trials.

Stroke patients in four NHS hospitals are now receiving genetic tests that determine whether a commonly prescribed drug will work for them – a breakthrough that could transform treatment for millions. Digital approaches are spearheading a drive to help make genomic medicine part of everyday care. The role of digital tools was a central theme at the HETT (Healthcare Excellence Through…

University of Stuttgart scientists develop enhanced CRISPR technique that makes genetic loss-of-function analyses more efficient and reproducible for medical research.

German researchers discover how HIV selects genome integration targets using RNA:DNA hybrids as guides, revealing new therapeutic approaches for controlling viral reservoirs.

A new study shows that a gene expression test on routinely collected prostate tissue can help guide therapy choices for prostate cancer patients whose cancer has spread to other parts of the body.

Chronic cough affects approximately 1 in 10 adults in the UK, but the causes are often unknown and treatment options limited. Now, a new study has identified neurological mechanisms as key drivers.