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

Schneider Y, Turan S, Koller A, Krumbiegel M, Farrell M, Plotz S, Winkler J, Xiang W (2022)

Publication Type: Journal article

Publication year: 2022

Journal

Stem Cell Research Elsevier

Book Volume: 65

DOI: 10.1016/j.scr.2022.102952

Abstract

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.

Authors with CRIS profile

Yanni Schneider Department of Molecular Neurology Aron Koller Department of Molecular Neurology Mandy Krumbiegel Institute of Human Genetics Jürgen Winkler Professur für Molekulare Neurologie Wei Xiang Department of Molecular Neurology

Additional Organisation(s)

Interdisziplinäres Zentrum für Klinische Forschung

Related research project(s)

E030: Th17 and beyond: immune-mediated neurotoxicity determines onset and progression in Parkinson’s disease April 1, 2020 - March 31, 2023 E030: Th17 and beyond: immune-mediated neurotoxicity determines onset and progression in Parkinson’s disease April 1, 2020 - March 31, 2023

How to cite

APA:

Schneider, Y., Turan, S., Koller, A., Krumbiegel, M., Farrell, M., Plotz, S.,... Xiang, W. (2022). 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. Stem Cell Research, 65. https://doi.org/10.1016/j.scr.2022.102952

MLA:

Schneider, Yanni, et al. "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." Stem Cell Research 65 (2022).

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