Health
Gene therapy to the inner retina prevents blindness in mouse model of CLN3 Batten disease – News-Medical.Net
Gene therapy to the inner retina prevented blindness in a mouse model of the neuro-degenerative disorder CLN3 Batten disease.
Reviewed by Emily Henderson, B.Sc.Aug 10 2020
Gene therapy to the inner retina prevented blindness in a mouse model of the neuro-degenerative disorder CLN3 Batten disease. Adeno-associated viral (AAV)-mediated expression of the human CLN3 gene led to significant survival of bipolar cells and pre-served retinal function, as reported in the peer-reviewed journal Human Gene Therapy.
Batten disease is a group of fatal, inherited lysosomal storage disorders that predominantly affect children. The m…
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