The DOI matches its Crossref record; 138 of the 138 references it cites were found in a registry or an index.
Delivery technologies for genome editing
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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In the citing paper
“The system can be designed to edit a large number of sequences in the genome with different sgRNAs and donor DNA templates, thus making it a powerful genome editing tool.[202, 205, 207]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [202]
In the citing paper
“The Cas9 nuclease utilizes a sgRNA to recognize the target sequence and subsequently cleaves the genome at the specific site.”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [202]
In the citing paper
“Viral delivery, such as lentivirus (LV) and adeno-associated virus (AAV), is also an effective method to deliver genome editing components.[202, 210]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [202]
In the citing paper
“These materials are similar to those used for mRNA delivery.[202, 205, 207–211]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [202]
Citations
Cites
Multiplex Genome Engineering Using CRISPR/Cas Systems
Cong, L., Ran, F. A., Cox, D., Lin, S., Barretto, R., Habib, N., Hsu, P. D., Wu, X., Jiang, W., Marraffini, L. A., et al. (2013) · Science · doi:10.1126/science.1231143 · Cited by 15,339
RNA-Guided Human Genome Engineering via Cas9
Mali, P., Yang, L., Esvelt, K. M., Aach, J., Guell, M., DiCarlo, J. E., Norville, J. E., & Church, G. M. (2013) · Science · doi:10.1126/science.1232033 · Cited by 9,210
The new frontier of genome engineering with CRISPR-Cas9
Doudna, J. A., & Charpentier, E. (2014) · Science · doi:10.1126/science.1258096 · Cited by 7,062
DNA targeting specificity of RNA-guided Cas9 nucleases
Hsu, P. D., Scott, D. A., Weinstein, J. A., Ran, F. A., Konermann, S., Agarwala, V., Li, Y., Fine, E. J., Wu, X., Shalem, O., et al. (2013) · Nature Biotechnology · doi:10.1038/nbt.2647 · Cited by 4,741
Webb, A. K. (2006) · Cited by 4,369
LMO2 -Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1
Hacein-Bey-Abina, S., Von Kalle, C., Schmidt, M., McCormack, M. P., Wulffraat, N., Leboulch, P., Lim, A., Osborne, C. S., Pawliuk, R., Morillon, E., et al. (2003) · Science · doi:10.1126/science.1088547 · Cited by 3,587
Wang, H., Yang, H., Shivalila, C. S., Dawlaty, M. M., Cheng, A. W., Zhang, F., & Jaenisch, R. (2013) · Cell · doi:10.1016/j.cell.2013.04.025 · Cited by 3,495
Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity
Ran, F. A., Hsu, P. D., Lin, C.-Y., Gootenberg, J. S., Konermann, S., Trevino, A. E., Scott, D. A., Inoue, A., Matoba, S., Zhang, Y., et al. (2013) · Cell · doi:10.1016/j.cell.2013.08.021 · Cited by 3,299
Non-viral vectors for gene-based therapy
Yin, H., Kanasty, R. L., Eltoukhy, A. A., Vegas, A. J., Dorkin, J. R., & Anderson, D. G. (2014) · Nature Reviews Genetics · doi:10.1038/nrg3763 · Cited by 3,123
Self-citationIn vivo genome editing using Staphylococcus aureus Cas9
Ran, F. A., Cong, L., Yan, W. X., Scott, D. A., Gootenberg, J. S., Kriz, A. J., Zetsche, B., Shalem, O., Wu, X., Makarova, K. S., et al. (2015) · Nature · doi:10.1038/nature14299 · Cited by 2,687
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Engineering precision nanoparticles for drug delivery
Mitchell, M. J., Billingsley, M. M., Haley, R. M., Wechsler, M. E., Peppas, N. A., & Langer, R. (2021) · Nature Reviews Drug Discovery · doi:10.1038/s41573-020-0090-8 · Cited by 7,933
Li, H., Yang, Y., Hong, W., Huang, M., Wu, M., & Zhao, X. (2020) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-019-0089-y · Cited by 1,829
Block copolymer micelles in nanomedicine applications
Cabral, H., Miyata, K., Osada, K., & Kataoka, K. (2018) · Chemical Reviews · doi:10.1021/acs.chemrev.8b00199 · Cited by 1,309
Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery
Kowalski, P. S., Rudra, A., Miao, L., & Anderson, D. G. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.02.012 · Cited by 1,127
Platt, F. M., d’Azzo, A., Davidson, B. L., Neufeld, E. F., & Tifft, C. J. (2018) · Nature Reviews Disease Primers · doi:10.1038/s41572-018-0025-4 · Cited by 1,052
Metadata
- 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.
- Venue
- Nature Reviews Drug Discovery
- Published
- 2017-03-24
- Volume
- 16
- Issue
- 6
- Pages
- 387-399
- ISSN
- 1474-1776 (ISSN-L)
- 1474-1784
- 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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