All-in-one adeno-associated virus delivery and genome editing by Neisseria meningitidis Cas9 in vivo.

ABSTRACT:

Background

Clustered, regularly interspaced, short palindromic repeats (CRISPR) and CRISPR-associated proteins (Cas) have recently opened a new avenue for gene therapy. Cas9 nuclease guided by a single-guide RNA (sgRNA) has been extensively used for genome editing. Currently, three Cas9 orthologs have been adapted for in vivo genome engineering applications: Streptococcus pyogenes Cas9 (SpyCas9), Staphylococcus aureus Cas9 (SauCas9), and Campylobacter jejuni (CjeCas9). However, additional in vivo editing platforms are needed, in part to enable a greater range of sequences to be accessed via viral vectors, especially those in which Cas9 and sgRNA are combined into a single vector genome.

Results

Here, we present in vivo editing using Neisseria meningitidis Cas9 (NmeCas9). Nme

SUBMITTER: Ibraheim R 

PROVIDER: S-EPMC6146650 | biostudies-literature | 2018 Sep

REPOSITORIES: biostudies-literature