By adding a microRNA molecule to ALS models, Tel Aviv University, Ben-Gurion University and Weizmann Institute researchers ...

In a first for adults in Singapore, scientists are conducting gene editing trials on heart patients to correct defects at their genetic source.

Earlier this year, researchers at CHOP and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a rare liver disorder. KJ Muldoon ...

Fred Hutch bioengineer Dr. Matthias Stephan is working to develop a special foam that could help make gene therapy for blood ...

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root cause of ...

A new international study led by Tel Aviv University researchers may pave the way for an effective treatment for amyotrophic ...

A rare disease that has cut off children from the outside world now potentially has a cure. A clinical trial to treat severe ...

As a new €2 million gene therapy research project funded by APC and Research Ireland is announced, we speak with the project lead Prof Niall Barron about its potential and how Ireland is positioning ...

Despite the best efforts of modern medicine, Huntington’s disease is a condition that still comes with a tragic prognosis.

We have written about the promise of gene therapy to correct genetic disease many times. An exciting development happened ...

Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to ...