Quality Control Strategy for CRISPR-Cas9-Based Gene Editing Complicated by a Pseudogene.

ABSTRACT: CRISPR-Cas9 mediated gene editing in induced pluripotent stem cells became an efficient tool to investigate biological mechanisms underlying genetic-driven diseases while accounting for the respective genetic background. This technique relies on the targeting of a specific nucleotide sequence present in the gene of interest. Therefore, the gene editing of some genes can be complicated by non-coding pseudogenes presenting a high homology of sequence with their respective genes. Among them, GBA is raising special interest because of its implication as the most common genetic risk factor for Parkinson's disease. In this study, we present an easy-to-use CRISPR-Cas9 gene editing strategy allowing for specific editing of point mutations in a gene without genetic alteration of its pseudogene exem

SUBMITTER: Hanss Z 

PROVIDER: S-EPMC6961559 | biostudies-literature | 2019

REPOSITORIES: biostudies-literature