gene therapy

Lab-Designed Virus Shows Promise for Inner Ear Gene Therapy

Delivery Method Could Eventually Help Correct Mutations That Cause Hearing Loss

By Brandon Levy

Tuesday, February 19, 2019

inner ear cells infected by a lab-designed virus with a gene that produces a glowing green protein

Most people probably think of viruses as villains that bring illnesses like measles, HIV, and the flu, but some viruses are proving to be valuable allies in the fight against genetic diseases. In a new study, a team of scientists from the NIH IRP and their colleagues showed the promise of a lab-designed virus for delivering gene therapies aimed at correcting hereditary hearing loss.

A Golden Injection for Christmas

By IRP Staff Blogger

Monday, January 11, 2016

Reblogged from The Children's Inn at NIH.

This past holiday season, 14-year old Gillian Copejans was the second person in the world to get an incredible gift: leading-edge gene-therapy treatment at the NIH Clinical Center.