NYSCF Innovator Feng Zhang Investigates the Role of Genetic Diversity in Future Gene Editing Treatments
The possibilities and implications of utilizing gene editing technology to treat patients raise thorny questions that must be addressed in a careful and thorough research and testing. NYSCF Robertson Stem Cell Investigator and NYSCF Robertson Stem Cell Prize recipient Dr. Feng Zhang, Broad Institute of Harvard and MIT, made an initial foray into addressing some of these possibilities, analyzing the implications of human genetic variation in CRISPR-based therapeutic genome editing.
Utilizing thousands of genome sets, Dr. Zhang determined that human genetic diversity led to rare but real instances confounding targets for gene editing, meaning the gene editing occurred in the incorrect spot due to normal and natural differences in the genetic code. This research, published in Nature Medicine, underscores the need for whole-genome sequencing as prescreening for patient candidates of future gene editing therapies to ensure safety and efficacy.