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
Summary
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 Dec;65:102952 |
| PubMed | 36283273 |
| DOI | 10.1016/j.scr.2022.102952 |