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

A Greer couple is hosting a fundraiser to support a costly gene editing procedure for their two children, who suffer from a ...

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.

Penn and CHOP researchers will utilize the new protocol to distribute customizable drugs that target specific genetic disorders.

Alzheimer's disease (AD) is a devastating cause of memory loss and cognitive decline, for which no curative treatment is available.

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

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 ...