Researchers identify genes to boost gene therapy efficiency

Researchers at the University of Wisconsin-Madison have developed a platform to screen the human genome to identify genes that, when silenced, improve the efficacy of gene therapies. After a six-year study, the team narrowed down a list of over 19,000 genes to two specific genes, GJB2 and BET1L.

Inactivating either of these two genes was found to increase the efficiency of base editing by more than six times in human cellular systems. This discovery could lead to new strategies, such as temporary gene deactivation, to enhance the performance of mRNA vaccines, immunotherapies, and CRISPR-Cas9 gene editing. The researchers have already tested this approach on retinal cells from patients with Leber congenital amaurosis.