Prevalence of Pre-existing Antibodies to CRISPR-Associated Nuclease Cas9 in the USA Population.

ABSTRACT: The repurposing of the CRISPR/Cas microbial adaptive immune system for gene editing has resulted in an exponential rise in new technologies and promising approaches for treating numerous human diseases. While some of the approaches being currently developed involve ex vivo editing by CRISPR/Cas9, many more potential applications will require in vivo editing. The in vivo use of this technology comes with challenges, one of which is the immune response to Cas9, a protein of microbial origin. Thus, the prevalence of pre-existing antibodies to Cas9 could also be a relevant parameter. There are many avenues for how CRISPR/Cas9 technologies will be applied in vivo, including the mode of delivery. These may be expected to invoke different immunological pathways. Noneth

SUBMITTER: Simhadri VL 

PROVIDER: S-EPMC6070699 | biostudies-literature | 2018 Sep

REPOSITORIES: biostudies-literature