CRISPR/Cas-Dependent and Nuclease-Free In Vivo Therapeutic Gene Editing.

ABSTRACT: Precise gene manipulation by gene editing approaches facilitates the potential to cure several debilitating genetic disorders. Gene modification stimulated by engineered nucleases induces a double-stranded break (DSB) in the target genomic locus, thereby activating DNA repair mechanisms. DSBs triggered by nucleases are repaired either by the nonhomologous end-joining or the homology-directed repair pathway, enabling efficient gene editing. While there are several ongoing ex vivo genome editing clinical trials, current research underscores the therapeutic potential of CRISPR/Cas-based (clustered regularly interspaced short palindrome repeats-associated Cas nuclease) in vivo gene editing. In this review, we provide an overview of the CRISPR/Cas-mediated in vivo genome th

SUBMITTER: Dasgupta I 

PROVIDER: S-EPMC7987363 | biostudies-literature | 2021 Mar

REPOSITORIES: biostudies-literature