Article
Developing a Gene Therapy for the Treatment of Autosomal Dominant Alzheimer's Disease.
Human gene therapy - 1 Oct 2023
Moore Benjamin, Sharma Apurwa, Goulet Martin, Suter Tracey, Pelletier Carolyn, Hu Ruoxi, Schaeffer Eric, Kelleher Raymond J
Abstract excerpt
Autosomal dominant Alzheimer's disease (ADAD) is a rare early-onset form of Alzheimer's disease, caused by dominant mutations in one of three genes: presenilin 1, presenilin 2, and amyloid β precursor protein (APP). Mutations in the presenilin 1 gene (PSEN1) account for the majority of cases, and individuals who inherit a single-mutant PSEN1 allele go on to develop early-onset dementia, ultimately leading to...
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