Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines.
Genetic medicines, including CRISPR/Cas technologies, extend tremendous promise for addressing unmet medical need in inherited retinal disorders and other indications; however, there remain challenges for the development of therapeutics. Herein, we evaluate genome editing by engineered Cas9 ribonucl...
Saved in:
Main Authors: | Spencer C Wei, Aaron J Cantor, Jack Walleshauser, Rina Mepani, Kory Melton, Ashil Bans, Prachi Khekare, Suhani Gupta, Jonathan Wang, Craig Soares, Radwan Kiwan, Jieun Lee, Shannon McCawley, Vihasi Jani, Weng In Leong, Pawan K Shahi, Jean Chan, Pierre Boivin, Peter Otoupal, Bikash R Pattnaik, David M Gamm, Krishanu Saha, Benjamin G Gowen, Mary Haak-Frendscho, Mary J Janatpour, Adam P Silverman |
---|---|
Format: | Article |
Language: | English |
Published: |
Public Library of Science (PLoS)
2025-01-01
|
Series: | PLoS ONE |
Online Access: | https://doi.org/10.1371/journal.pone.0317387 |
Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
Similar Items
-
Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines
by: Spencer C. Wei, et al.
Published: (2025-01-01) -
Industrialization in Indonesia : developments and prospects /
by: McCawley, Peter
Published: (1979) -
Thirty million theories of grammar
by: McCawley, James D.
Published: (1982) -
Clinical and laboratory characteristics of patients with systemic sclerosis positive for anti-ribonucleoprotein antibodies
by: R. U. Shayakhmetova, et al.
Published: (2019-11-01) -
Mesenchymal stem cells with magnetic particles for the subretinal injection in ophthalmology
by: Yu. A. Beliy, et al.
Published: (2014-07-01)