Cited by 597CRISPR and Genetic EngineeringRNA Interference and Gene DeliveryVirus-based gene therapy research

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Delivery technologies for genome editing

Yin, Hao, Kauffman, Kevin J., Anderson, Daniel G.·2017·Nature Reviews Drug Discovery

DOI 10.1038/nrd.2016.280

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Abstract

With the recent development of CRISPR technology, it is becoming increasingly easy to engineer the genome. Genome-editing systems based on CRISPR, as well as transcription activator-like effector nucleases (TALENs) and zinc-finger nucleases (ZFNs), are becoming valuable tools for biomedical research, drug discovery and development, and even gene therapy. However, for each of these systems to effectively enter cells of interest and perform their function, efficient and safe delivery technologies are needed. This Review discusses the principles of biomacromolecule delivery and gene editing, examines recent advances and challenges in non-viral and viral delivery methods, and highlights the status of related clinical trials.

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Authors: Yin, H., Kauffman, K. J., & Anderson, D. G.
Affiliations: Koch Institute for Integrative Cancer Research At MIT (US)
Harvard–MIT Division of Health Sciences and Technology (US)
Corresponding author: Anderson, Daniel G.
ORCID: Yin, Hao
Kauffman, Kevin J.
Anderson, Daniel G.
Venue: Nature Reviews Drug Discovery
Publisher: Springer Science and Business Media LLC
Published: 2017-03-24
Volume: 16
Issue: 6
Pages: 387-399
ISSN: 1474-1776 (ISSN-L)
1474-1784
DOI: 10.1038/nrd.2016.280
OpenAlex: W2598091501
Cited by: 597
References: 138
Citations by year: 2026: 41 · 2025: 56 · 2024: 65 · 2023: 59 · 2022: 64 · 2021: 63 · 2020: 84 · 2019: 61 · 2018: 81 · 2017: 22
FWCI: 6.34
Type: journal article
Language: en
Open access: closed
Licence: www.springer.com/tdm (tdm)
Locations: Nature Reviews Drug Discovery (publishedVersion)
PubMed (publishedVersion)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Keywords: genome editing, biomacromolecule delivery, CRISPR, TALEN, zinc finger nucleases, non-viral delivery, viral vectors, gene therapy, clinical trials, intracellular delivery
MeSH terms: Transcription Activator-Like Effector Nucleases, Gene Editing, Animals, Genetic Engineering, Humans, Genetic Therapy, Genome, Human, Zinc Fingers, Gene Transfer Techniques, Biomedical Research, Drug Discovery, Clustered Regularly Interspaced Short Palindromic Repeats

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