CRISPR-mediated Ophthalmic Genome Surgery

Galaxy Y Cho; Yazeed Abdulla; Jesse D Sengillo; Sally Justus; Kellie A Schaefer; Alexander G Bassuk; Stephen H Tsang; Vinit B Mahajan

doi:10.1007/s40135-017-0144-1

Journal article

CRISPR-mediated Ophthalmic Genome Surgery

Galaxy Y Cho, Yazeed Abdulla, Jesse D Sengillo, Sally Justus, Kellie A Schaefer, Alexander G Bassuk, Stephen H Tsang and Vinit B Mahajan

Current ophthalmology reports, Vol.5(3), pp.199-206

09/2017

DOI: 10.1007/s40135-017-0144-1

PMCID: PMC5613978

PMID: 28966884

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https://www.ncbi.nlm.nih.gov/pmc/articles/5613978View

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Abstract

Clustered regularly interspaced short palindromic repeats (CRISPR) is a genome engineering system with great potential for clinical applications due to its versatility and programmability. This review highlights the development and use of CRISPR-mediated ophthalmic genome surgery in recent years.\nDiverse CRISPR techniques are in development to target a wide array of ophthalmic conditions, including inherited and acquired conditions. Preclinical disease modeling and recent successes in gene editing suggest potential efficacy of CRISPR as a therapeutic for inherited conditions. In particular, the treatment of Leber congenital amaurosis with CRISPR-mediated genome surgery is expected to reach clinical trials in the near future.\nTreatment options for inherited retinal dystrophies are currently limited. CRISPR-mediated genome surgery methods may be able to address this unmet need in the future.

CRISPR-Cas

induced pluripotent stem cells

genome surgery

inherited retinal dystrophy

Details

Title: Subtitle: CRISPR-mediated Ophthalmic Genome Surgery
Creators: Galaxy Y Cho - Department of Pathology & Cell Biology, Institute of Human Nutrition, College of Physicians and Surgeons, Columbia University, New York, NY, USA
Yazeed Abdulla - School of Medicine, The University of Jordan, Amman, Jordan
Jesse D Sengillo - State University of New York Downstate Medical Center, Brooklyn, NY, USA
Sally Justus - Jonas Children's Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University Medical Center, New York, NY, USA
Kellie A Schaefer - Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, USA
Alexander G Bassuk - Department of Pediatrics and Neurology, University of Iowa, Iowa City, IA, USA
Stephen H Tsang - Jonas Children's Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University Medical Center, New York, NY, USA
Vinit B Mahajan - Department of Ophthalmology, Byers Eye Institute, Stanford University, Palo Alto, CA, USA
Resource Type: Journal article
Publication Details: Current ophthalmology reports, Vol.5(3), pp.199-206
DOI: 10.1007/s40135-017-0144-1
PMID: 28966884
PMCID: PMC5613978
NLM abbreviation: Curr Ophthalmol Rep
ISSN: 2167-4868
eISSN: 2167-4868
Publisher: United States
Grant note: R01 EY025225 / NEI NIH HHS\nR01 EY018213 / NEI NIH HHS\nP30 CA013696 / NCI NIH HHS\nR01 EY024698 / NEI NIH HHS\nR01 EY026682 / NEI NIH HHS\nR01 EY024665 / NEI NIH HHS\nF31 EY026789 / NEI NIH HHS\nR21 AG050437 / NIA NIH HHS\nP30 EY019007 / NEI NIH HHS
Language: English
Date published: 09/2017
Academic Unit: Neurology; Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Neurology (Pediatrics)
Record Identifier: 9984070332002771

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