Precision genome editing in the eye-Reference-Cited by-同舟云学术

Precision genome editing in the eye

Published:2022-09-19 Issue:39 Volume:119 Page:
ISSN:0027-8424
Container-title:Proceedings of the National Academy of Sciences
language:en
Short-container-title:Proc. Natl. Acad. Sci. U.S.A.

Author:

Suh Susie¹^ORCID,Choi Elliot H.¹^ORCID,Raguram Aditya²³⁴,Liu David R.²³⁴,Palczewski Krzysztof¹⁵⁶⁷^ORCID

Affiliation:

1. Gavin Herbert Eye Institute, Department of Ophthalmology, University of California, Irvine, CA 92697

2. Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA 02142

3. Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA 02138

4. Howard Hughes Medical Institute, Harvard University, Cambridge, MA 02138

5. Department of Physiology and Biophysics, University of California, Irvine, CA 92697

6. Department of Chemistry, University of California, Irvine, CA 92697

7. Department of Molecular Biology and Biochemistry, University of California, Irvine, CA 92697

Abstract

CRISPR-Cas-based genome editing technologies could, in principle, be used to treat a wide variety of inherited diseases, including genetic disorders of vision. Programmable CRISPR-Cas nucleases are effective tools for gene disruption, but they are poorly suited for precisely correcting pathogenic mutations in most therapeutic settings. Recently developed precision genome editing agents, including base editors and prime editors, have enabled precise gene correction and disease rescue in multiple preclinical models of genetic disorders. Additionally, new delivery technologies that transiently deliver precision genome editing agents in vivo offer minimized off-target editing and improved safety profiles. These improvements to precision genome editing and delivery technologies are expected to revolutionize the treatment of genetic disorders of vision and other diseases. In this Perspective, we describe current preclinical and clinical genome editing approaches for treating inherited retinal degenerative diseases, and we discuss important considerations that should be addressed as these approaches are translated into clinical practice.

Funder

HHS | NIH | National Eye Institute

HHS | NIH | National Institute of General Medical Sciences

Division of Intramural Research, National Institute of Allergy and Infectious Diseases

HHS | NIH | National Human Genome Research Institute

Publisher

Proceedings of the National Academy of Sciences

Subject

Multidisciplinary

Link

https://pnas.org/doi/pdf/10.1073/pnas.2210104119

Reference88 articles.