Advances in Care

The Gene Messenger: Advancing Neurological Gene Therapy

The Gene Messenger: Advancing Neurological Gene Therapy
The Gene Messenger: Advancing Neurological Gene Therapy

On Monday, August 18th, 2003 Dr. Michael Kaplitt administered the first trial of neurological gene therapy in a human patient. In a procedure that almost didn’t happen, he used adeno-associated virus to deliver a gene into the brain of a patient with Parkinson’s. The results were groundbreaking and set new avenues of research in neurological gene therapy in motion. In this episode, Dr. Kaplitt tells the story of this breakthrough, and what he’s working on next to continue evolving the field of neurosurgery.

After establishing that neurological gene therapy can help address brain cells damaged by Parkinson’s, Dr. Michael Kaplitt and his team have set their sights on a new mission: preventative intervention. For patients with an inherited form of Parkinson’s caused by a GBA gene mutation, Dr. Kaplitt and his colleagues are working to deliver genes that can stop the degeneration of damaged braincells, as well as initiating the use of focused ultrasound to treat essential tremors. In this episode, Dr. Kaplitt details the mechanisms of his work and the advancements he’s working on today.