Journal
CELL
Volume 181, Issue 1, Pages 136-150Publisher
CELL PRESS
DOI: 10.1016/j.cell.2020.03.023
Keywords
-
Categories
Funding
- Pitt Hopkins Research Foundation
- Grace Science Foundation
- NIH [1R01-HG009761, 1R01-MH110049, 1DP1-HL141201, 5R-M1HG006193-09]
- Howard Hughes Medical Institute
- Harold G. and Leila Mathers, Jr. Foundation
- Edward Mallinckrodt, Jr. Foundation
- Poitras Center for Psychiatric Disorders Research
- Hock E. Tan and K. Lisa Yang Center for Autism Research at MIT
- Open Philanthropy Project, the Phillips family
- NIH
- Cystic Fibrosis Foundation
- Michelson Found Animals Foundation
- Believe in a Cure
Ask authors/readers for more resources
The development of clustered regularly interspaced short-palindromic repeat (CRISPR)-based biotechnologies has revolutionized the life sciences and introduced new therapeutic modalities with the potential to treat a wide range of diseases. Here, we describe CRISPR-based strategies to improve human health, with an emphasis on the delivery of CRISPR therapeutics directly into the human body using adeno-associated virus (AAV) vectors. We also discuss challenges facing broad deployment of CRISPR-based therapeutics and highlight areas where continued discovery and technological development can further advance these revolutionary new treatments.
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available