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Prime Editing for the Installation and Correction of Mutations Causing Inherited Retinal Disease: A Brief Methodology.
- Source :
-
Methods in molecular biology (Clifton, N.J.) [Methods Mol Biol] 2023; Vol. 2560, pp. 313-331. - Publication Year :
- 2023
-
Abstract
- 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 <superscript>rd10</superscript> c.1678C > T (p.Arg560Cys) mutation which causes autosomal recessive retinitis pigmentosa (RP) in mice.<br /> (© 2023. The Author(s), under exclusive license to Springer Science+Business Media, LLC, part of Springer Nature.)
- Subjects :
- Mice
Animals
Proof of Concept Study
Mutation
Retinal Diseases
Subjects
Details
- Language :
- English
- ISSN :
- 1940-6029
- Volume :
- 2560
- Database :
- MEDLINE
- Journal :
- Methods in molecular biology (Clifton, N.J.)
- Publication Type :
- Academic Journal
- Accession number :
- 36481907
- Full Text :
- https://doi.org/10.1007/978-1-0716-2651-1_29