Gene Editing Core Facility

Core Director: Eric Kmiec, Ph.D.

The technological progress of human gene editing directed by CRISPR/Cas9 is quickly becoming a legitimate developmental tool for human therapeutics. The new gene editing core facility will aid in the translation of bench top research into clinical and bedside arenas by assisting in the design and syntheses of gene editing tools to function with improved accuracy for the treatment of Sickle Cell Disease and other inherited diseases. Providing expertise in developing clinical strategies of translation to the COBRE grant.

Throughout Dr. Kmiec’s professional career, he has led research teams studying the reaction mechanics, biochemistry and molecular genetics of gene editing in human cells. His early work with sickle cell disease led to research and development of the next generation of gene editing tools, including CRISPRs (clustered regularly interspaced short palindromic repeats) and to even more promising variations such as single-stranded DNA oligonucleotides (ssODNs) for the treatment of inherited disorders.
He holds 18 issued patents, most of which have been licensed by biotechnology and pharmaceutical companies and has founded two biotechnology companies. He is a senior scientific advisor and SAB member of ETAGEN, a gene editing company located in Cambridge, Massachusetts, centered on the development of therapeutic uses of gene editing.

Dr. Kmiec has been the primary mentor for 18 Ph.D. students and 4 MS students, all of whom have attained scientific positions. He has held or holds major administrative posts on various NIH regional and state biomedical research grants, including IDeA Network of Biomedical Research (INBRE) and Centers of Biomedical Research Excellence (COBRE). Dr. Kmiec was also honored as the Eminent Scholar in residence at Marshall University (Huntington, West Virginia) in 2009-2011 and was elected as an Honorary Commander of the 436th Air Wing at Dover Air Force Base in Dover, Delaware for in 2013 and again in 2014.

He is also the founder of The Gene Editing Institute (GEI) in the Helen F. Graham Cancer Center and Research Institute in 2015.

Publications: https://www.ncbi.nlm.nih.gov/myncbi/browse/collection/48079036/?sort=date&direction=ascending