Ontology highlight
ABSTRACT:
SUBMITTER: Li F
PROVIDER: S-EPMC7511709 | biostudies-literature | 2020
REPOSITORIES: biostudies-literature
Li Fan F Wing Kristof K Wang Jiang-Hui JH Luu Chi D CD Bender James A JA Chen Jinying J Wang Qi Q Lu Qinyi Q Nguyen Tran Minh Thuan MT Young Kaylene M KM Wong Raymond C B RCB Pébay Alice A Cook Anthony L AL Hung Sandy S C SSC Liu Guei-Sheung GS Hewitt Alex W AW
Frontiers in cellular neuroscience 20200910
CRISPR/Cas has opened the prospect of direct gene correction therapy for some inherited retinal diseases. Previous work has demonstrated the utility of adeno-associated virus (AAV) mediated delivery to retinal cells <i>in vivo</i>; however, with the expanding repertoire of CRISPR/Cas endonucleases, it is not clear which of these are most efficacious for retinal editing <i>in vivo</i>. We sought to compare CRISPR/Cas endonuclease activity using both single and dual AAV delivery strategies for gen ...[more]