Connor Ludwig (University of California, San Francisco)
Martin Kampmann (University of California, San Francisco)
Rulin Tian (University of California, San Francisco)
This invention includes human induced pluripotent stem cell (iPSC) lines that harbor a single copy dCas9-BFP-KRAB at the CLYBL safe harbor locus (mediating CRISPR inhibition of human gene expression) and/or a single copy of dox-inducible NGN2 at the AAVS1 locus (enabling the differentiation of the iPSCs into neurons). The CRISPR-mediated inhibition of human gene expression is maintained into the differentiated neurons, permitting functional studies of targeted genes in neurons.
The stem cell lines can be used for forward genetic and chemogenetic screens for drug tests of neurological diseases.
The stem cell lines included in this technology permit direct genetic and screening approaches on neurons.