Therapeutic Gene Editing in Inherited Retinal Disorders

Jinjie Ling; Laura A Jenny; Ashley Zhou; Stephen H Tsang

doi:10.1101/cshperspect.a041292

Therapeutic Gene Editing in Inherited Retinal Disorders

Cold Spring Harb Perspect Med. 2023 Apr 3;13(4):a041292. doi: 10.1101/cshperspect.a041292.

Authors

Jinjie Ling¹, Laura A Jenny², Ashley Zhou¹, Stephen H Tsang^{3

4

5

6}

Affiliations

¹ Columbia University Vagelos College of Physicians and Surgeons, New York, New York 10032, USA.
² Jonas Children's Vision Care, and Bernard and Shirley Brown Glaucoma Laboratory, Edward Harkness Eye Institute, Department of Ophthalmology, New York-Presbyterian Hospital, New York, New York 10032, USA.
³ Jonas Children's Vision Care, and Bernard and Shirley Brown Glaucoma Laboratory, Edward Harkness Eye Institute, Department of Ophthalmology, New York-Presbyterian Hospital, New York, New York 10032, USA sht2@columbia.edu.
⁴ Department of Biomedical Engineering, Columbia University, New York, New York 10032, USA.
⁵ Columbia Stem Cell Initiative, and Institute of Human Nutrition, Columbia University, New York, New York 10032, USA.
⁶ Department of Pathology and Cell Biology, Columbia University, New York, New York 10032, USA.

PMID: 36096547
PMCID: PMC10071418 (available on 2025-04-01)
DOI: 10.1101/cshperspect.a041292

Abstract

Since the development of CRISPR/Cas9 gene editing in 2012, therapeutic editing research has produced several phase 1-2a trials. Here we provide an overview of the mechanisms and applications of various gene-editing technologies including adeno-associated virus vectors, lentiviruses, CRISPR/Cas9 systems, base and prime editing, antisense oligonucleotides, short-hairpin RNAs, Cas13, and adenosine deaminase acting on RNA for the treatment of various inherited retinal diseases (IRDs). We outline the various stages of clinical trials using these technologies and the impacts they have made in advancing the practice of medicine.

Publication types

Review
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't

MeSH terms

CRISPR-Cas Systems
Gene Editing*
Humans
Retinal Diseases* / genetics
Retinal Diseases* / therapy

Abstract

Publication types

MeSH terms

Grants and funding