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 

Advanced Search

close
  • Publications
  • Application Notes
  • Posters
  • Workshops
  • Videos & Webinars
  • Articles
Search

Browse by Category

  • Application
    • Diagnostic and Imaging
    • Genetic Medicine
    • Hematology
    • Metabolic Disorders
    • Neuroscience
    • Oncology
    • Skeletal Disorders
    • Targeted Drug Delivery
    • Vaccines
    • Other Applications
    • Cell therapy
  • Formulation
    • Liposomes
    • Nucleic Acid Lipid Nanoparticles
    • Polymeric Nanoparticles
    • Other Formulations
  • Payload
    • DNA
    • microRNA
    • mRNA
    • siRNA
    • Small Molecule Drugs
    • Other Payloads


related content

Publication - Summary

The Cellular Mechanisms of Neuronal Swelling Underlying Cytotoxic Edema

R.L. Rungta, H.B. Choi, J.R. Tyson, A. Malik, L. Dissing-Olesen, P.J.C. Lin, S.M. Cain, P.R. Cullis, T.P. Snutch and B.A. MacVicar

In this paper, the MacVicar Lab used Neuro9 siRNA cell transfection nanoparticles (Neuro9-siRNA) prepared using the NanoAssemblrTM Benchtop instrument to uncover the role of a specific chloride channel in cytotoxic edema, which is a...

Read More


Publication - Abstract

Systemic delivery of RNA interference (RNAi) payloads for manipulation of gene expression in lymphocytes holds a great potential as a novel therapeutic modality for hematological malignancies and autoimmune disorders. However, lymphocytes are among the most difficult cells to tra...
Read More


Sign Up and Stay Informed
Sign up today to automatically receive new Precision NanoSystems application notes, conference posters, relevant science publications, and webinar invites.