Application of CRISPR-Cas9 in eye disease

Wenyi Wu; Luosheng Tang; Patricia A D'Amore; Hetian Lei

doi:10.1016/j.exer.2017.06.007

Application of CRISPR-Cas9 in eye disease

Exp Eye Res. 2017 Aug:161:116-123. doi: 10.1016/j.exer.2017.06.007. Epub 2017 Jun 12.

Authors

Wenyi Wu¹, Luosheng Tang², Patricia A D'Amore³, Hetian Lei⁴

Affiliations

¹ Schepens Eye Research Institute of Massachusetts Eye and Ear, Department of Ophthalmology, Harvard Medical School, Boston, MA 02114, United States; Department of Ophthalmology, Second Xiangya Hospital, Central South University, Changsha, Hunan, China.
² Department of Ophthalmology, Second Xiangya Hospital, Central South University, Changsha, Hunan, China.
³ Schepens Eye Research Institute of Massachusetts Eye and Ear, Department of Ophthalmology, Harvard Medical School, Boston, MA 02114, United States; Department of Pathology, Harvard Medical School, Boston, MA 02114, United States.
⁴ Schepens Eye Research Institute of Massachusetts Eye and Ear, Department of Ophthalmology, Harvard Medical School, Boston, MA 02114, United States. Electronic address: hetian_lei@meei.harvard.edu.

PMID: 28619505
DOI: 10.1016/j.exer.2017.06.007

Abstract

The system of clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated nuclease (Cas)9 is an effective instrument for revising the genome with great accuracy. This system has been widely employed to generate mutants in genomes from plants to human cells. Rapid improvements in Cas9 specificity in eukaryotic cells have opened great potential for the use of this technology as a therapeutic. Herein, we summarize the recent advancements of CRISPR-Cas9 use in research on human cells and animal models, and outline a basic and clinical pipeline for CRISPR-Cas9-based treatments of genetic eye diseases.

Publication types

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

MeSH terms

Animals
CRISPR-Cas Systems / genetics*
Eye Diseases / therapy*
Gene Targeting
Genetic Therapy*
Humans
RNA Editing*

Grants and funding

R01 EY012509/EY/NEI NIH HHS/United States