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Literature DB >> 30783971

Use of AAV Vectors for CRISPR-Mediated In Vivo Genome Editing in the Retina.

Abstract

Degenerative retinal diseases such as retinitis pigmentosa (RP) and Leber's congenital amaurosis (LCA) may lead to blindness without effective treatment. With the rapid advancement of the CRISPR/Cas9 genome editing technology, in vivo application of CRISPR/Cas9 holds immense potential for treatment of these diseases. Adeno-associated virus (AAV) vectors are an ideal gene transfer tool for delivery of CRISPR components to the retina. Here, we describe a protocol for utilizing an AAV-based CRISPR/Cas9 system for in vivo genome editing in the retina.

Entities: CellLine Chemical Disease Gene Mutation Species

Keywords: AAV vector; CRISPR/Cas9; Gene therapy; Genome editing; Retina; Retinal degeneration

Mesh：

Substances：
RNA, Guide

Year: 2019 PMID： 30783971 PMCID： PMC6730636 DOI： 10.1007/978-1-4939-9139-6_7

Source DB: PubMed Journal: Methods Mol Biol ISSN： 1064-3745

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Cited

7 in total

1. Gene Therapy Approaches to Slow or Reverse Blindness From Inherited Retinal Degeneration: Growth Factors and Optogenetics.

Authors: Russell N Van Gelder
Journal: Int Ophthalmol Clin Date: 2021-10-01

Review 2. Development of clustered regularly interspaced short palindromic repeats/CRISPR-associated technology for potential clinical applications.

Authors: Yue-Ying Huang; Xiao-Yu Zhang; Ping Zhu; Ling Ji
Journal: World J Clin Cases Date: 2022-06-26 Impact factor: 1.534

Use of AAV Vectors for CRISPR-Mediated In Vivo Genome Editing in the Retina.

1. Gene Therapy Approaches to Slow or Reverse Blindness From Inherited Retinal Degeneration: Growth Factors and Optogenetics.

Review 2. Development of clustered regularly interspaced short palindromic repeats/CRISPR-associated technology for potential clinical applications.

3. Antiproliferative effects of AAV-delivered CRISPR/Cas9-based degradation of the HPV18-E6 gene in HeLa cells.

Review 4. Nanocarriers: A novel strategy for the delivery of CRISPR/Cas systems.

5. Inhibition of Hepatitis B Virus by AAV8-Derived CRISPR/SaCas9 Expressed From Liver-Specific Promoters.

6. Efficient CRISPR editing with a hypercompact Cas12f1 and engineered guide RNAs delivered by adeno-associated virus.

7. Restoration of RPGR expression in vivo using CRISPR/Cas9 gene editing.