Generation of two induced pluripotent stem cell lines and the corresponding isogenic controls from Parkinson's disease patients carrying the heterozygous mutations c.1290A > G (p.T351A) or c.2067A > G (p.T610A) in the RHOT1 gene encoding Miro1


Primary skin fibroblasts from two Parkinson's disease (PD) patients carrying distinct heterozygous mutations in the RHOT1 gene encoding Miro1, namely c.1290A > G (Miro1 p.T351A) and c.2067A > G (Miro1 p.T610A), were converted into induced pluripotent stem cells (iPSCs) by episomal reprogramming. The corresponding isogenic gene-corrected lines have been generated using CRISPR/Cas9 technology. Here, we provide a comprehensive characterization and quality assurance of both isogenic pairs, which will be used to study Miro1-related molecular mechanisms underlying neurodegeneration in iPSC-derived neuronal models (e.g., midbrain dopaminergic neurons and astrocytes). Copyright © 2023 The Authors. Published by Elsevier B.V. All rights reserved.

Authors Chemla A, Arena G, Saraiva C, Berenguer-Escuder C, Grossmann D, Grünewald A, Klein C, Seibler P, Schwamborn JC, Krüger R
Journal Stem cell research
Publication Date 2023 Jun;69:103085
PubMed 37003181
PubMed Central PMC10240566
DOI 10.1016/j.scr.2023.103085

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