Stem Cell Research (Jul 2019)

A collection of integration-free iPSCs derived from Parkinson's disease patients carrying mutations in the GBA1 gene

  • Eva Rodríguez-Traver,
  • Eva Díaz-Guerra,
  • César Rodríguez,
  • Pablo Fernández,
  • Fabián Arenas,
  • Marcos Araúzo-Bravo,
  • María Orera,
  • Jaime Kulisevsky,
  • Rosario Moratalla,
  • Carlos Vicario

Journal volume & issue
Vol. 38

Abstract

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Mutations in the GBA1 gene, which encodes the lysosomal enzyme Glucocerebrosidase1 are major risk factors for Parkinson's disease (PD) and dementia with Lewy bodies (DLB). We have generated induced pluripotent stem cells (iPSCs) from fibroblasts of four PD patients carrying the N370S/wt or the L444P/wt heterozygous mutations in GBA1. The iPSCs presented a normal morphology, expressed endogenous pluripotency markers and could be differentiated into endodermal, mesodermal and ectodermal cells. These iPSCs were free from Sendai viral vectors and reprogramming factors, had a normal karyotype and maintained the original GBA1 genotype.