Journal article
CRISPR GENOME SURGERY IN THE RETINA IN LIGHT OF OFF-TARGETING
Retina (Philadelphia, Pa.), Vol.38(8), pp.1443-1455
08/2018
DOI: 10.1097/IAE.0000000000002197
PMCID: PMC6054556
PMID: 29746416
Abstract
Recent concerns regarding the clinical utilization of clustered regularly interspaced short palindromic repeats (CRISPR) involve uncertainties about the potential detrimental effects that many arise due to unintended genetic changes, as in off-target mutagenesis, during CRISPR genome surgery. This review gives an overview of off-targeting detection methods and CRISPR's place in the clinical setting, specifically in the field of ophthalmology.
As CRISPR utilization in the laboratory setting has increased, knowledge regarding CRISPR mechanisms including its off-target effects has also increased. Although a perfect method for achieving 100% specificity is yet to be determined, the past few years have seen many developments in off-targeting detection and in increasing efficacy of CRISPR tools.
The CRISPR system has high potential to be an invaluable therapeutic tool as it has the ability to modify and repair pathogenic retinal lesions. Although it is not yet a perfect system, with further efforts to improve its specificity and efficacy along with careful screening of off-target mutations, CRISPR-mediated genome surgery potential can become maximized and applied to patients.
Details
- Title: Subtitle
- CRISPR GENOME SURGERY IN THE RETINA IN LIGHT OF OFF-TARGETING
- Creators
- Galaxy Y Cho - Department of Ophthalmology, Columbia University, New York, New YorkKellie A Schaefer - Department of Ophthalmology, Omics Lab, Byers Eye Institute, Stanford University, Palo Alto, CaliforniaAlexander G Bassuk - Department of Pediatrics, University of Iowa, Iowa City, IowaStephen H Tsang - Department of Cell Biology and Pathology, Columbia University, New York, New YorkVinit B Mahajan - Palo Alto Veterans Administration, Palo Alto, California
- Resource Type
- Journal article
- Publication Details
- Retina (Philadelphia, Pa.), Vol.38(8), pp.1443-1455
- DOI
- 10.1097/IAE.0000000000002197
- PMID
- 29746416
- PMCID
- PMC6054556
- NLM abbreviation
- Retina
- ISSN
- 0275-004X
- eISSN
- 1539-2864
- Publisher
- United States
- Grant note
- R01 EY025225 / NEI NIH HHS R01 EY018213 / NEI NIH HHS P30 CA013696 / NCI NIH HHS R01 EY024698 / NEI NIH HHS R01 EY026682 / NEI NIH HHS R01 EY024665 / NEI NIH HHS F31 EY026789 / NEI NIH HHS R21 AG050437 / NIA NIH HHS P30 EY019007 / NEI NIH HHS
- Language
- English
- Date published
- 08/2018
- Academic Unit
- Neurology; Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Neurology (Pediatrics)
- Record Identifier
- 9984070572102771
Metrics
38 Record Views