Journal article
CRISPR-Cas9 genome engineering: Treating inherited retinal degeneration
Progress in Retinal and Eye Research, Vol.65, pp.28-49
07/2018
DOI: 10.1016/j.preteyeres.2018.03.003
PMCID: PMC8210531
PMID: 29578069
Abstract
Gene correction is a valuable strategy for treating inherited retinal degenerative diseases, a major cause of irreversible blindness worldwide. Single gene defects cause the majority of these retinal dystrophies. Gene augmentation holds great promise if delivered early in the course of the disease, however, many patients carry mutations in genes too large to be packaged into adeno-associated viral vectors and some, when overexpressed via heterologous promoters, induce retinal toxicity. In addition to the aforementioned challenges, some patients have sustained significant photoreceptor cell loss at the time of diagnosis, rendering gene replacement therapy insufficient to treat the disease. These patients will require cell replacement to restore useful vision. Fortunately, the advent of induced pluripotent stem cell and CRISPR-Cas9 gene editing technologies affords researchers and clinicians a powerful means by which to develop strategies to treat patients with inherited retinal dystrophies. In this review we will discuss the current developments in CRISPR-Cas9 gene editing in vivo in animal models and in vitro in patient-derived cells to study and treat inherited retinal degenerative diseases.
Details
- Title: Subtitle
- CRISPR-Cas9 genome engineering: Treating inherited retinal degeneration
- Creators
- Erin R Burnight - Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, United StatesJoseph C Giacalone - Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, United StatesJessica A Cooke - University of Iowa, The University of Iowa Institute for Vision ResearchJessica R Thompson - Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, United StatesLaura R Bohrer - Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, United StatesKathleen R Chirco - Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, United StatesArlene V Drack - Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, United StatesJohn H Fingert - Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, United StatesKristan S Worthington - Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, United StatesLuke A Wiley - Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, United StatesRobert F Mullins - Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, United StatesEdwin M Stone - Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, United StatesBudd A Tucker - Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, United States
- Resource Type
- Journal article
- Publication Details
- Progress in Retinal and Eye Research, Vol.65, pp.28-49
- DOI
- 10.1016/j.preteyeres.2018.03.003
- PMID
- 29578069
- PMCID
- PMC8210531
- NLM abbreviation
- Prog Retin Eye Res
- ISSN
- 1350-9462
- eISSN
- 1873-1635
- Publisher
- Elsevier Ltd
- Language
- English
- Date published
- 07/2018
- Academic Unit
- Roy J. Carver Department of Biomedical Engineering; Stead Family Department of Pediatrics; The University of Iowa Institute for Vision Research; Iowa Neuroscience Institute; Chemical and Biochemical Engineering; Ophthalmology and Visual Sciences
- Record Identifier
- 9983980053502771
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