July 07, 2017
Efficient and safe delivery of the CRISPR/Cas system is one of the key challenges for genome-editing applications in humans. Herein, we designed and synthesized a series of biodegradable lipidlike compounds containing ester groups for the delivery of mRNA-encoding Cas9. Two lead materials, termed N-methyl-1,3-propanediamine (MPA)-A and MPA-Ab, showed a tunable rate of biodegradation. MPA-A with linear ester chains was degraded dramatically faster than MPA-Ab with branched ester chains in the presence of esterase or in wild-type mice. Most importantly, MPA-A and MPA-Ab demonstrated efficient delivery of Cas9 mRNA both in vitro and in vivo. Consequently, these biodegradable lipidlike nanomaterials merit further development as genome-editing delivery tools for biological and therapeutic
Publication - Abstract
Sep 24, 2019
Nature Communications
Publication - Summary
Apr 27, 2017
Molecular Therapy
The swine influenza pandemic of 2009 and more recent zoonotic transmissions of several avian influenza subtypes to human populations punctuates the threat posed by new pathogens for which we have no pre-existing immunity. Traditional vaccines requi...