The DOI matches its Crossref record; 337 of the 337 references it cites were found in a registry or an index.
Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects
Open accessAbstract
Based on engineered or bacterial nucleases, the development of genome editing technologies has opened up the possibility of directly targeting and modifying genomic sequences in almost all eukaryotic cells. Genome editing has extended our ability to elucidate the contribution of genetics to disease by promoting the creation of more accurate cellular and animal models of pathological processes and has begun to show extraordinary potential in a variety of fields, ranging from basic research to applied biotechnology and biomedical research. Recent progress in developing programmable nucleases, such as zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs) and clustered regularly interspaced short palindromic repeat (CRISPR)–Cas-associated nucleases, has greatly expedited the progress of gene editing from concept to clinical practice. Here, we review recent advances of the three major genome editing technologies (ZFNs, TALENs, and CRISPR/Cas9) and discuss the applications of their derivative reagents as gene editing tools in various human diseases and potential future therapies, focusing on eukaryotic cells and animal models. Finally, we provide an overview of the clinical trials applying genome editing platforms for disease treatment and some of the challenges in the implementation of this technology.
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Citations
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Cited by
CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis
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Targeting the RAS/RAF/MAPK pathway for cancer therapy: from mechanism to clinical studies
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Metadata
- Authors
- Li, H., Yang, Y., Hong, W., Huang, M., Wu, M., & Zhao, X.
- Venue
- Signal Transduction and Targeted Therapy
- Published
- 2020-01-03
- Volume
- 5
- Issue
- 1
- Pages
- 1-1
- ISSN
- 2059-3635 (ISSN-L)
- OpenAlex
- W2998881619
- Cited by
- 1,715
- References
- 337
- FWCI
- 73.09
- Type
- journal article
- Language
- en
- Open access
- gold
- Licence
- creativecommons.org/licenses/by/4.0 (tdm)
- creativecommons.org/licenses/by/4.0 (vor)
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