Generation of a homozygous and a heterozygous SNCA gene knockout human-induced pluripotent stem cell line by CRISPR/Cas9 mediated allele-specific tuning of SNCA expression


Aggregation of alpha-synuclein (aSyn) is closely linked to Parkinson's disease, probably due to the loss of physiological functions and/or gain of toxic functions of aggregated aSyn. Significant efforts have been made elucidating the physiological structure and function of aSyn, however, with limited success thus far in human-derived cells, partly because of restricted resources. Here, we developed two human-induced pluripotent stem cell lines using CRISPR/Cas9-mediated allele-specific frame-shift deletion of the aSyn encoding gene SNCA, resulting in homo- and heterozygous SNCA knockout. The generated cell lines are promising cellular tools for studying aSyn dosage-dependent functions and structural alterations in human neural cells. Copyright © 2022 The Authors. Published by Elsevier B.V. All rights reserved.

Authors Schneider Y, Turan S, Koller A, Krumbiegel M, Farrell M, Plötz S, Winkler J, Xiang W
Journal Stem cell research
Publication Date 2022 Oct 20;65:102952
PubMed 36283273
DOI 10.1016/j.scr.2022.102952

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