Details

Autor(en) / Beteiligte

• Lee, Seo-Young
• Chung, Sun-Ku

Titel

Generation of gene-corrected iPSC line, KIOMi002-A, from Parkinson's disease patient iPSC with LRRK2 G2019S mutation using BAC-based homologous recombination

Ist Teil von

• Stem cell research, 2019-12, Vol.41, p.101649-101649, Article 101649

Ort / Verlag

England: Elsevier B.V

Erscheinungsjahr

2019

Quelle

Elsevier ScienceDirect Journals Complete

Beschreibungen/Notizen

• •We genetically corrected the LRRK2 G2019S gene mutation.•A BAC-based homologous recombination system was used as a gene replacement tool.•Gene-corrected iPSCs had the characteristics of pluripotent stem cells. Mutations in leucine-rich repeat kinase 2 (LRRK2) gene (LRRK2 G2019S) is a representative autosomal dominant mutation that can cause Parkinson's disease (PD). A bacterial artificial chromosome-based homologous recombination (BAC-based HR) system was utilized for gene therapy of LRRK2 G2019S-mutant induced pluripotent stem cells (iPSCs) produced by reprogramming episomal vectors. The gene-corrected iPSCs retained typical pluripotency required for their spontaneous differentiation into differentiated cells. The iPSCs had a normal karyotype and were confirmed to have no off-target sites by melting curve analysis.