Article
Prime Editing for the Installation and Correction of Mutations Causing Inherited Retinal Disease: A Brief Methodology.
Methods in molecular biology (Clifton, N.J.) - 1 Jan 2023
Tsai Yi-Ting, da Costa Bruna Lopes, Nolan Nicholas D, Caruso Salvatore Marco, Jenny Laura A, Levi Sarah R, Tsang Stephen H, Quinn Peter M J
Abstract excerpt
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...
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