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Mary Ann Liebert: Gene Therapy to Treat Mucopolysaccharidosis IVA
October 31, 2024
NEW ROCHELLE, New York, Oct. 31 (TNSres) -- Mary Ann Liebert Inc. issued the following news release:

Reporting in the peer-reviewed journal Human Gene Therapy, researchers used ex vivo lentiviral gene therapy to treat mucopolysaccharidosis IVA (MPS IVA) in mice. Click here [View article in the link at bottom.] to read the article now.

MPS IVA is an autosomal recessive disease caused by a mutation in the N-acetylgalactosamine-5-sulfate sulfatase (GALNS) gene resulting in . . .

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