Article
AAVrh.10 delivery of novel APOE2-Christchurch variant suppresses amyloid and Tau pathology in Alzheimer's disease mice.
Molecular therapy : the journal of the American Society of Gene Therapy - 4 Dec 2024
Günaydin Caner, Sondhi Dolan, Kaminsky Stephen M, Lephart Hailey C, Leopold Philip L, Hackett Neil R, Khanna Richie, Crystal Ronald G
Abstract excerpt
Gene therapy to treat hereditary disorders conventionally delivers the normal allele to compensate for loss-of-function mutations. More effective gene therapy may be achieved using a gain-of-function variant. We tested the hypothesis that AAVrh.10-mediated CNS delivery of the human APOE2 allele with the Christchurch mutation (R136S) (E2Ch) will provide superior protection against APOE4-associated Alzheimer's...
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