A CRISPR/Cas9-based system for reprogramming cell lineage specification.

TitleA CRISPR/Cas9-based system for reprogramming cell lineage specification.
Publication TypeJournal Article
Year of Publication2014
AuthorsS Chakraborty, H Ji, AM Kabadi, CA Gersbach, N Christoforou, and KW Leong
JournalStem Cell Reports
Start Page940
Pagination940 - 947
Date Published12/2014

Gene activation by the CRISPR/Cas9 system has the potential to enable new approaches to science and medicine, but the technology must be enhanced to robustly control cell behavior. We show that the fusion of two transactivation domains to Cas9 dramatically enhances gene activation to a level that is necessary to reprogram cell phenotype. Targeted activation of the endogenous Myod1 gene locus with this system led to stable and sustained reprogramming of mouse embryonic fibroblasts into skeletal myocytes. The levels of myogenic marker expression obtained by the activation of endogenous Myod1 gene were comparable to that achieved by overexpression of lentivirally delivered MYOD1 transcription factor.

Short TitleStem Cell Reports