Combining Engineered Nucleases with Adeno-associated Viral Vectors for Therapeutic Gene Editing

Overview

Journal Adv Exp Med Biol

Specialties Biology
General Medicine

Date 2017 Nov 14

PMID 29130152

Citations 8

Authors

Benjamin E Epstein

David V Schaffer

Affiliations

Soon will be listed here.

Abstract

With the recent advent of several generations of targeted DNA nucleases, most recently CRISPR/Cas9, genome editing has become broadly accessible across the biomedical community. Importantly, the capacity of these nucleases to modify specific genomic loci associated with human disease could render new classes of genetic disease, including autosomal dominant or even idiopathic disease, accessible to gene therapy. In parallel, the emergence of adeno-associated virus (AAV) as a clinically important vector raises the possibility of integrating these two technologies towards the development of gene editing therapies. Though clear challenges exist, numerous proof-of-concept studies in preclinical models offer exciting promise for the future of gene therapy.

Citing Articles

Efforts to Downsize Base Editors for Clinical Applications.

Song B Int J Mol Sci. 2025; 26(5).

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Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working Group.

Cavazza A, Molina-Estevez F, Plaza Reyes A, Ronco V, Naseem A, Malensek S Mol Ther Nucleic Acids. 2025; 36(1):102457.

PMID: 39991472 PMC: 11847086. DOI: 10.1016/j.omtn.2025.102457.

Beyond the promise: evaluating and mitigating off-target effects in CRISPR gene editing for safer therapeutics.

Lopes R, Prasad M Front Bioeng Biotechnol. 2024; 11:1339189.

PMID: 38390600 PMC: 10883050. DOI: 10.3389/fbioe.2023.1339189.

Efficient DNA knock-in using AAV-mediated delivery with 2-cell embryo CRISPR-Cas9 electroporation.

Davis D, McNew J, Maresca-Fichter H, Chen K, Telugu B, Bryda E Front Genome Ed. 2023; 5:1256451.

PMID: 37694158 PMC: 10485772. DOI: 10.3389/fgeed.2023.1256451.

The Role of Recombinant AAV in Precise Genome Editing.

Bijlani S, Pang K, Sivanandam V, Singh A, Chatterjee S Front Genome Ed. 2022; 3:799722.

PMID: 35098210 PMC: 8793687. DOI: 10.3389/fgeed.2021.799722.

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