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publicationDate 2022-03-10-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber US-2022072151-A1
titleOfInvention Methods and Gene Therapy Constructs for Treating GM2 Gangliosidoses
abstract Disclosed are novel gene therapy constructs containing both HEXA and HEXB genes to treat GM2 gangliosidoses, including Sandhoff disease and Tay-Sach's disease. Also described are co-treatments using chaperone and anti-inflammatory agents to enhance the effects of gene therapy.
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