CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors

Overview

Journal Cell

Publisher Cell Press

Specialty Cell Biology

Date 2020 Apr 4

PMID 32243786

Citations 217

Authors

Dan Wang

Feng Zhang

Guangping Gao

Affiliations

Soon will be listed here.

Abstract

The development of clustered regularly interspaced short-palindromic repeat (CRISPR)-based biotechnologies has revolutionized the life sciences and introduced new therapeutic modalities with the potential to treat a wide range of diseases. Here, we describe CRISPR-based strategies to improve human health, with an emphasis on the delivery of CRISPR therapeutics directly into the human body using adeno-associated virus (AAV) vectors. We also discuss challenges facing broad deployment of CRISPR-based therapeutics and highlight areas where continued discovery and technological development can further advance these revolutionary new treatments.

Citing Articles

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Adeno-Associated Virus Vectors: Principles, Practices, and Prospects in Gene Therapy.

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In vivo targeted gene delivery using Adenovirus-antibody molecular glue conjugates.

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gene editing using primary cells derived from Cas9-expressing pigs.

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