Repurposing Gene Therapy for Batten Disease

Completed Research, Dr. Crystal, Neuro, Other, Pediatric, Rare, Weill Cornell Medical College

Principal Investigator: Dr. Ron Crystal

Disease: Batten Disease

Research Description: Batten Diseases comprise a group of genetic diseases that affect children. Children with these gene defects are missing key enzymes that help to break down waste products in brain cells. These waste products build up, and eventually destroy the brain cells. This causes a loss of function in these children, and eventually death. Dr. Crystal and his team are undertaking a pilot clinical trial to determine the impact of putting a working copy of the affected gene into a virus that can then be injected into the brain. These brain cells would produce the missing enzyme, allowing the brain cells to survive and the children to thrive. If successful, this study could produce a life-saving treatment for children with Batten Disease.

Funding Partners: Anonymous

CWR funding role: Primary funder

Completed: 2018

Completed Research