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

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 ...

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 ...

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

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

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.

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

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

In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...

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 ...