advertisement
Abstract #106606 Published in IGR 23-3
Prime Editing for the Installation and Correction of Mutations Causing Inherited Retinal Disease: A Brief Methodology
Tsai YT; da Costa BL; Nolan ND; Caruso SM; Caruso SM; Jenny LA; Levi SR; Tsang SH; Quinn PMJ; Quinn PMJ; Quinn PMJMethods in molecular biology (Clifton, N.J.) 2023; 2560: 313-331
Inherited retinal diseases (IRDs) encompass a large heterogeneous group of rare blinding disorders whose etiology originates from mutations in the 280 genes identified to date. Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) systems represent a promising avenue for the treatment of IRDs, as exemplified by FDA clinical trial approval of EDIT-101 (AGN-151587), which removes a deep intronic variant in the CEP290 gene that causes Leber congenital amaurosis (LCA) type 10. Prime editing is a novel double-strand break (DSB) independent CRISPR/Cas system which has the potential to correct all 12 possible transition and transversion mutations in addition to small deletions and insertions. Here, as a proof-of-concept study, we describe a methodology using prime editing for the in vitro installation and correction of the classical Pde6b c.1678C > T (p.Arg560Cys) mutation which causes autosomal recessive retinitis pigmentosa (RP) in mice.
Columbia University, Department of Biomedical Engineering, New York, NY, USA.
Full article