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

Researchers report early but encouraging results on an experimental gene-editing treatment for high cholesterol.

For more than a decade, the strongest AIDS drugs could not fully control Matt Chappell's HIV infection. Now his body controls it by itself, and researchers are trying to perfect the gene editing that ...

The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy ...

The approach is “very exciting,” but one expert says a lot more research is needed, especially around safety, before clinical ...

In a world first, a bespoke gene-editing therapy benefited one child. Now researchers plan to launch a clinical trial of the ...

The year 2023 marked a significant milestone for the treatment of sickle cell disease (SCD), with the approval of two gene therapies shown to help reduce the pain experienced by people with this ...

MeiraGTx is cashing in on a gene therapy that restored vision to 11 children who were born legally blind, licensing the ...

FOLLY BEACH — His pale blue eyes flashing and sporting a wide grin, Max Harbin toddled forward, leaning on a small cart as he ventured a few steps. His parents, Jim and Laura Harbin, watched him ...

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

The FDA has restricted the number of patients who can receive the Cambridge drugmaker's gene therapy for Duchenne muscular ...