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Article

In vivo genome editing in animals using AAV-CRISPR system: applications to translational research of human disease

2017-12-20

Abstract excerpt

Adeno-associated virus (AAV) has shown promising therapeutic efficacy with a good safety profile in a wide range of animal models and human clinical trials. With the advent of clustered regulatory interspaced short palindromic repeat (CRISPR)-based genome-editing technologies, AAV provides one of the most suitable viral vectors to package, deliver, and express CRISPR components for targeted gene editing. Recent di...

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Literature Corpus work
3fa31f31-3a08-5c66-9dcc-0f7843543e5e
DOI
10.12688/f1000research.11243.1
Open publication

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