Unknown

Dataset Information

0

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.

SUBMITTER: Yu W 

PROVIDER: S-EPMC6730636 | biostudies-literature | 2019

REPOSITORIES: biostudies-literature

Json Xml
altmetric image

Publications

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

Yu Wenhan W   Wu Zhijian Z  

Methods in molecular biology (Clifton, N.J.) 20190101

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/  ...[more]

Similar Datasets

Unknown CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors.
| S-EPMC7236621 | biostudies-literature
Unknown A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing.
| S-EPMC6313841 | biostudies-other
Unknown CRISPR/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice.
| S-EPMC5482879 | biostudies-other
Unknown AAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria.
| S-EPMC6962637 | biostudies-literature
Unknown Factors Impacting Efficacy of AAV-Mediated CRISPR-Based Genome Editing for Treatment of Choroidal Neovascularization.
| S-EPMC7044682 | biostudies-literature
Unknown CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors.
| S-EPMC5568673 | biostudies-literature
Unknown Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo.
| S-EPMC8560862 | biostudies-literature
Unknown CRISPR/Cpf1-mediated DNA-free plant genome editing.
| S-EPMC5316869 | biostudies-other
Unknown In vivo genome editing in animals using AAV-CRISPR system: applications to translational research of human disease.
| S-EPMC5749125 | biostudies-literature
Unknown In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration.
| S-EPMC5331785 | biostudies-literature