Generation of isogenic control DJ-1-delP GC13 for the genetic Parkinson's disease-patient derived iPSC line DJ-1-delP (LCSBi008-A-1)

为遗传性帕金森病患者衍生的 iPSC 系 DJ-1-delP (LCSBi008-A-1) 生成同源对照 DJ-1-delP GC13

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作者:Pauline Mencke, Zoé Hanss, Javier Jarazo, François Massart, Arkadiusz Rybicki, Elizabet Petkovski, Enrico Glaab, Ibrahim Boussaad, Vincenzo Bonifati, Jens Christian Schwamborn, Wim Mandemakers, Rejko Krüger

Abstract

We describe the generation of an isogenic control cell line DJ-1-delP GC13 from an induced pluripotent stem cell (iPSC) line DJ-1-delP LCSBi008-A that was derived from fibroblasts obtained from a Parkinson's disease (PD) patient. Using CRISPR/Cas9 technology, we corrected the disease causing c.471_473delGCC homozygous mutation in the PARK7 gene leading to p.158P deletion in the encoded protein DJ-1. The generated isogenic pair will be used for phenotypic analysis of PD-patient derived neurons and astrocytes.

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