Article ID Journal Published Year Pages File Type
5522615 Stem Cell Research 2017 5 Pages PDF
Abstract

We describe the generation of a biallelic GBA deletion human embryonic stem cell line using zinc finger nuclease-mediated gene targeting. The homozygous targeting of exon 4 of the GBA locus leads to a complete loss of glucocerebrosidase (GCase) protein expression.

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Life Sciences Biochemistry, Genetics and Molecular Biology Biotechnology