Multistage Delivery Nanoparticle Facilitates Efficient CRISPR/dCas9 Activation and Tumor Growth Suppression In Vivo.

ABSTRACT: CRISPR/dCas9 systems can precisely control endogenous gene expression without interrupting host genomic sequence and have provided a novel and feasible strategy for the treatment of cancers at the transcriptional level. However, development of CRISPR/dCas9-based anti-cancer therapeutics remains challenging due to the conflicting requirements for the design of the delivery system: a cationic and membrane-binding surface facilitates the tumor accumulation and cellular uptake of the CRISPR/dCas9 system, but hinders the circulating stability in vivo. Here, a multistage delivery nanoparticle (MDNP) that can achieve tumor-targeted delivery of CRISPR/dCas9 systems and restore endogenous microRNA (miRNA) expression in vivo is described. MDNP is designed as a core-shell structure in which the shell

SUBMITTER: Liu Q 

PROVIDER: S-EPMC6325604 | biostudies-literature | 2019 Jan

REPOSITORIES: biostudies-literature