Title : Targeting Gys1 with AAV-SaCas9 Decreases Pathogenic Polyglucosan Bodies and Neuroinflammation in Adult Polyglucosan Body and Lafora Disease Mouse Models.

Pub. Date : 2021 Apr

PMID : 33830476






1 Functional Relationships(s)
Download
Sentence
Compound Name
Protein Name
Organism
1 We packaged Staphylococcus aureus Cas9 and a guide RNA targeting the glycogen synthase gene, Gys1, responsible for brain glycogen branch elongation in AAV9 virus, which we delivered by neonatal intracerebroventricular injection to one mouse model of adult polyglucosan body disease and two mouse models of Lafora disease. Glycogen glycogen synthase 1, muscle Mus musculus