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

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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Bibliographic Details
Main Authors: Evangelos Konstantinidis, Agnieszka Molisak, Florian Perrin, Linn Streubel-Gallasch, Sarah Fayad, Daniel Y. Kim, Karl Petri, Martin J. Aryee, Ximena Aguilar, Bence György, Vilmantas Giedraitis, J. Keith Joung, Vikram Pattanayak, Magnus Essand, Anna Erlandsson, Oksana Berezovska, Martin Ingelsson
Format: Article
Language:English
Published: Elsevier 2022-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT:: RNA/DNA editing
Alzheimer’s disease
presenilin 1
amyloid-β
fibroblasts
CRISPR-Cas9
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253122000713

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