A CRISPR-based gene therapy showed improvement in lipids in difficult-to-treat patients, but one study raised flags after a death last month in a trial of an unrelated gene therapy.

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

In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol ...

An experimental gene-editing therapy developed by Crispr Therapeutics is showing promise for treating heart disease.

Gene therapy is a revolutionary approach to treating genetic disorders by introducing functional genes into cells to replace or correct defective genes. It involves the use of genetic material, such ...

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment ...

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 ...

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

While gene therapy for cystic fibrosis is still in the research phase, researchers aim to correct the defective gene responsible for the disease. Current research is promising. Cystic fibrosis (CF) is ...