期刊
ADVANCED DRUG DELIVERY REVIEWS
卷 168, 期 -, 页码 181-195出版社
ELSEVIER
DOI: 10.1016/j.addr.2020.06.011
关键词
CRISPR/Cas; Genome editing; Gene therapy; Gene delivery; AAV vector; Retinal degeneration
资金
- Intramural Research Program of the National Eye Institute at the National Institutes of Health [1ZIAEY000443-12]
- NATIONAL EYE INSTITUTE [ZIAEY000443] Funding Source: NIH RePORTER
Inherited or multifactorial ocular diseases require novel treatment paradigms, with CRISPR genome editing technology showing great promise. However, the delivery of CRISPR/Cas components to target ocular tissues and cells must consider safety and efficacy. Viral and non-viral vectors are used for delivery, but sustained expression of CRISPR/Cas may lead to immune reactions and off-target editing risks.
A variety of inherited or multifactorial ocular diseases call for novel treatment paradigms. The newly developed genome editing technology, CRISPR, has shown great promise in treating these diseases, but delivery of the CRISPR/Cas components to target ocular tissues and cells requires appropriate use of vectors and routes of administration to ensure safety, efficacy and specificity. Although adeno-associated viral (AAV) vectors are thus far the most commonly used tool for ocular gene delivery, sustained expression of CRISPR/Cas components may cause immune reactions and an increased risk of off-target editing. In this review, we summarize the ocular administration routes and discuss the advantages and disadvantages of viral and non-viral vectors for delivery of CRISPR/Cas components to the eye. We review the existing studies of CRISPR/Cas genome editing for ocular diseases and discuss the major challenges of the technology in ocular applications. We also discuss the most recently developed CRISPR tools such as base editing and prime editing which may be used for future ocular applications. Published by Elsevier B.V.
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