A biodegradable nanocapsule delivers a Cas9 ribonucleoprotein complex for in vivo genome editing.

ABSTRACT: Delivery technologies for the CRISPR-Cas9 (CRISPR, clustered regularly interspaced short palindromic repeats) gene editing system often require viral vectors, which pose safety concerns for therapeutic genome editing¹. Alternatively, cationic liposomal components or polymers can be used to encapsulate multiple CRISPR components into large particles (typically >100 nm diameter); however, such systems are limited by variability in the loading of the cargo. Here, we report the design of customizable synthetic nanoparticles for the delivery of Cas9 nuclease and a single-guide RNA (sgRNA) that enables the controlled stoichiometry of CRISPR components and limits the possible safety concerns in vivo. We describe the synthesis of a thin glutathione (GSH)-cleavable covalently crosslinked

SUBMITTER: Chen G 

PROVIDER: S-EPMC6778035 | biostudies-literature | 2019 Oct

REPOSITORIES: biostudies-literature