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

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

The Food and Drug Administration is adding a new warning to a gene therapy linked to two patient deaths earlier this year.

In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn ...

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

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

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

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

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

Krystal Biotech drives Vyjuvek growth, premium margins, global expansion, a $1B goal, and pipeline data ahead with a $219.50 ...

Cincinnati Children's Hospital has dedicated a new state-of-the-art facility for applied gene and cell therapy in Sharonville ...