Charge-altering releasable transporters (CARTs) for the delivery and release of mRNA in living animals.

ABSTRACT: Functional delivery of mRNA to tissues in the body is key to implementing fundamentally new and potentially transformative strategies for vaccination, protein replacement therapy, and genome editing, collectively affecting approaches for the prevention, detection, and treatment of disease. Broadly applicable tools for the efficient delivery of mRNA into cultured cells would advance many areas of research, and effective and safe in vivo mRNA delivery could fundamentally transform clinical practice. Here we report the step-economical synthesis and evaluation of a tunable and effective class of synthetic biodegradable materials: charge-altering releasable transporters (CARTs) for mRNA delivery into cells. CARTs are structurally unique and operate through an unprecedented mechanism, serving in

SUBMITTER: McKinlay CJ 

PROVIDER: S-EPMC5278438 | biostudies-literature | 2017 Jan

REPOSITORIES: biostudies-literature