Promising gene therapy delivers treatment directly to brain
The Associated Press
Scientists say delivering gene therapy directly to the brain holds great promise.
The first brain-delivered gene therapy on the market was recently approved in Europe and the U.K. for a rare genetic disorder called AADC deficiency.
It causes developmental delays and movement disorders in kids.
New Jersey drugmaker PTC Therapeutics plans to seek approval for the treatment in the U.S. this year.
Meanwhile, about 30 studies in the U.S. are testing gene therapy directly to the brain for other disorders, including Alzheimer’s and Parkinson’s.
Challenges remain, especially with diseases caused by more than a single gene. But scientists say the evidence supporting this approach is mounting.
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1/24/2023 3:49:41 PM (GMT -6:00)