Mary Ann Liebert Inc.: New AAV9 Gene Therapy Vector Dramatically Increases Life Span in Krabbe Disease Mouse Model
August 12, 2019
August 12, 2019
NEW ROCHELLE, New York, Aug. 12 -- Mary Ann Liebert Inc. issued the following news release:
An optimized and newly engineered form of the adeno-associated vector 9 (AAV9) vector used to deliver the galactosylceramidase gene to a mouse model of the inherited neurogenerative and rapidly fatal form of Krabbe disease improved clinical symptoms and prolonged median survival by 275%. Two-day old mice treated with a single injection of the systemic gene therapy had a significant increase i . . .
An optimized and newly engineered form of the adeno-associated vector 9 (AAV9) vector used to deliver the galactosylceramidase gene to a mouse model of the inherited neurogenerative and rapidly fatal form of Krabbe disease improved clinical symptoms and prolonged median survival by 275%. Two-day old mice treated with a single injection of the systemic gene therapy had a significant increase i . . .