CRISPR-Cas9 treatment partially restores amyloid-β 42/40 in human fibroblasts with the Alzheimer’s disease PSEN1 M146L mutation

Presenilin 1 (PS1) is a central component of γ-secretase, an enzymatic complex involved in the generation of the amyloid-β (Aβ) peptide that deposits as plaques in the Alzheimer’s disease (AD) brain. The M146L mutation in the PS1 gene (PSEN1) leads to an autosomal dominant form of early-onset AD by...

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Detalles Bibliográficos
Autores principales: Konstantinidis, Evangelos, Molisak, Agnieszka, Perrin, Florian, Streubel-Gallasch, Linn, Fayad, Sarah, Kim, Daniel Y., Petri, Karl, Aryee, Martin J., Aguilar, Ximena, György, Bence, Giedraitis, Vilmantas, Joung, J. Keith, Pattanayak, Vikram, Essand, Magnus, Erlandsson, Anna, Berezovska, Oksana, Ingelsson, Martin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9043867/
https://www.ncbi.nlm.nih.gov/pubmed/35505961
http://dx.doi.org/10.1016/j.omtn.2022.03.022

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