Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...

University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a mouse ...

Alzheimer’s may be driven far more by genetics than previously thought, with one gene playing an outsized role. Researchers ...

Future GLP-1 gene therapy could enable long-term hormone production, with Fractyl Health and others planning human trials in 2024.

SEATTLE — Some of our most challenging health conditions result from genetic disorders like sickle cell disease and certain cancers. Gene therapy is revolutionizing how they're treated, and the Fred ...

Gazi University in Ankara has officially launched the production process for Türkiye's first local and national gene therapy research product. The project, which targets rare genetic diseases, is ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages. Today, we can intervene in the first days or weeks of life.

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

A research team at the University of California San Diego has discovered a novel and promising method of treating arrhythmogenic cardiomyopathy (ACM), a rare inherited heart disease that can strike ...

Qatar set to be among top five best countries in precision health revolutionLifestyle, behaviour changes can help ‘turn on’ g ...

After telegraphing an investigation in November, the FDA has moved to restrict the use of bluebird bio’s gene therapy Skysona in certain patients. The FDA has updated Skysona’s indication, allowing it ...