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

An experimental gene-editing treatment shows promise for permanently lowering levels of cholesterol and triglycerides, ...

Until now, researchers assumed that schizophrenia, anxiety disorders or depression arise from an interplay of many different ...

Promising results from a small clinical trial highlight a growing interest in designing gene-editing treatments for common ...

One dose of an experimental drug cover offer lifetime treatment for people with high cholesterol, but its long-term safety is ...

The real revolution came in 2012, when researchers Jennifer Doudna and Emmanuelle Charpentier harnessed CRISPR, a natural bacterial defense system. In bacteria, CRISPR cuts out invading viruses’ DNA ...

The FDA added a boxed warning to delandistrogene moxeparvovec (Elevidys) gene therapy for Duchenne muscular dystrophy and ...

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

By reactivating a long-lost gene, researchers were able to lower uric acid levels and stop damaging fat accumulation in human ...

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

It must be aggressive enough to fight infections and cancer, yet restrained enough to avoid attacking the body's own tissues.