Functionalized lipid-like nanoparticles for in vivo mRNA delivery and base editing

Author:

Zhang Xinfu12ORCID,Zhao Weiyu1ORCID,Nguyen Giang N.3ORCID,Zhang Chengxiang1,Zeng Chunxi1ORCID,Yan Jingyue1,Du Shi1,Hou Xucheng1,Li Wenqing1,Jiang Justin1,Deng Binbin4,McComb David W.45ORCID,Dorkin Robert6,Shah Aalok6ORCID,Barrera Luis6ORCID,Gregoire Francine6,Singh Manmohan6,Chen Delai6ORCID,Sabatino Denise E.37ORCID,Dong Yizhou1891011ORCID

Affiliation:

1. Division of Pharmaceutics and Pharmacology, College of Pharmacy, The Ohio State University, Columbus, OH 43210, USA.

2. State Key Laboratory of Fine Chemicals, Dalian University of Technology, Dalian 116024, China.

3. The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.

4. Center for Electron Microscopy and Analysis, Department of Materials Science and Engineering, The Ohio State University, Columbus, OH 43210, USA.

5. Department of Materials Science and Engineering, The Ohio State University, Columbus, OH 43210, USA.

6. Beam Therapeutics, Cambridge, MA 02139, USA.

7. Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.

8. Department of Biomedical Engineering, The Ohio State University, Columbus, OH 43210, USA.

9. The Center for Clinical and Translational Science, The Ohio State University, Columbus, OH 43210, USA.

10. The Comprehensive Cancer Center, The Ohio State University, Columbus, OH 43210, USA.

11. Dorothy M. Davis Heart and Lung Research Institute, The Ohio State University, Columbus, OH 43210, USA.

Abstract

Biodegradable nanomaterials effectively deliver long messenger RNAs in animal models.

Funder

National Heart, Lung, and Blood Institute

National Institute of General Medical Sciences

Publisher

American Association for the Advancement of Science (AAAS)

Subject

Multidisciplinary

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