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

• DOI: 10.1021/acsami.7b08163
• Publication Date: 2017-07-07T09:15:21Z
• Authors: Xinfu Zhang; Bin Li; Xiao Luo; Weiyu Zhao; Justin Jiang; Chengxiang Zhang; Min Gao; Xiaofang Chen; Yizhou Dong
• Keywords: Nanomaterials; Esterase

Efficient and safe delivery of the CRISPR/Cas system is one key challenges for genome-editing applications in humans. Herein, we designed synthesized a series biodegradable lipidlike compounds containing ester groups mRNA-encoding Cas9. Two lead materials, termed N-methyl-1,3-propanediamine (MPA)-A MPA-Ab, showed tunable rate biodegradation. MPA-A with linear chains was degraded dramatically faster than MPA-Ab branched presence esterase or wild-type mice. Most importantly, demonstrated efficient Cas9 mRNA both vitro vivo. Consequently, these nanomaterials merit further development as tools biological therapeutic applications.