The laboratories of Dr. Kevin Bender and Dr. Nadav Ahituv at the University of California, San Francisco, today announced the publication of preclinical proof-of-concept data demonstrating that CRISPR activation restores SCN2A expression and ameliorates disease-related phenotypes in mouse and human models of SCN2A-haploinsufficiency. The study, published today in Nature, represents a major advance for the treatment of SCN2A-haploinsufficiency, one of the most common genetic causes of neurodevelopmental disorders and early-onset epilepsy.
Congratulations to doctors Nadav Ahituv and Kevin Bender!