Biodegradable Amino-ester Nanomaterials for Cas9 mRNA Delivery in Vitro and in Vivo


Authors: X. Zhang, B. Li, X. Luo, W. Zhao, J. Jiang, C. Zhang, M. Gao, X. Chen, and Y. Dong

Journal: ACS Applied Materials & Interfaces

DOI: 10.1021/acsami.7b08163

Publication - Abstract

July 07, 2017

Abstract:

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 

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