The DOI matches its Crossref record; 41 of the 41 references it cites were found in a registry or an index.
Translation of CRISPR Genome Surgery to the Bedside for Retinal Diseases
Abstract
In recent years, there has been accelerated growth of clustered regularly interspaced short palindromic repeats (CRISPR) genome surgery techniques. Genome surgery holds promise for diseases for which a cure currently does not exist. In the field of ophthalmology, CRISPR offers possibilities for treating inherited retinal dystrophies. The retina has little regenerative potential, which makes treatment particularly difficult. For such conditions, CRISPR genome surgery methods have shown great potential for therapeutic applications in animal models of retinal dystrophies. Much anticipation surrounds the potential for CRISPR as a therapeutic, as clinical trials of ophthalmic genome surgery are expected to begin as early as 2018. This mini-review summarizes preclinical CRISPR applications in the retina and current CRISPR clinical trials.
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“To date, CRISPR/Cas9 has been used to develop animal models, understand disease mechanisms, and validate treatment targets [2,3,4,5].”
From Viral Delivery Systems for CRISPR (2019) · cites this paper as [3]
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Cites
1 retracted
Unexpected mutations after CRISPR–Cas9 editing in vivo
Schaefer, K. A., Wu, W.-H., Colgan, D. F., Tsang, S. H., Bassuk, A. G., & Mahajan, V. B. (2017) · Nature Methods · doi:10.1038/nmeth.4293 · Cited by 286
Self-citationRetractedMultiplex 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
GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases
Tsai, S. Q., Zheng, Z., Nguyen, N. T., Liebers, M., Topkar, V. V., Thapar, V., Wyvekens, N., Khayter, C., Iafrate, A. J., Le, L. P., et al. (2015) · Nature Biotechnology · doi:10.1038/nbt.3117 · Cited by 2,268
RNA-programmed genome editing in human cells
Jinek, M., East, A., Cheng, A., Lin, S., Ma, E., & Doudna, J. (2013) · eLife · doi:10.7554/elife.00471 · Cited by 2,098
In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration
Suzuki, K., Tsunekawa, Y., Hernandez-Benitez, R., Wu, J., Zhu, J., Kim, E. J., Hatanaka, F., Yamamoto, M., Araoka, T., Li, Z., et al. (2016) · Nature · doi:10.1038/nature20565 · Cited by 1,150
Defining and improving the genome-wide specificities of CRISPR–Cas9 nucleases
Tsai, S. Q., & Joung, J. K. (2016) · Nature Reviews Genetics · doi:10.1038/nrg.2016.28 · Cited by 510
Directed evolution using dCas9-targeted somatic hypermutation in mammalian cells
Hess, G. T., Frésard, L., Han, K., Lee, C. H., Li, A., Cimprich, K. A., Montgomery, S. B., & Bassik, M. C. (2016) · Nature Methods · doi:10.1038/nmeth.4038 · Cited by 476
The role of the PD‐1 pathway in autoimmunity and peripheral tolerance
Fife, B. T., & Pauken, K. E. (2011) · Annals of the New York Academy of Sciences · doi:10.1111/j.1749-6632.2010.05919.x · Cited by 355
Research works printed but not linked: 1
- Taylor, 2017, Editas delays IND for Allergan-Partnered CRISPR Program
Cited by
Viral Delivery Systems for CRISPR
Xu, C. L., Ruan, M. Z. C., Mahajan, V. B., & Tsang, S. H. (2019) · Viruses · doi:10.3390/v11010028 · Cited by 277
Self-citationRNA-targeting strategies as a platform for ocular gene therapy
Kumar, S., Fry, L. E., Wang, J.-H., Martin, K. R., Hewitt, A. W., Chen, F. K., & Liu, G.-S. (2023) · Progress in Retinal and Eye Research · doi:10.1016/j.preteyeres.2022.101110 · Cited by 51
Metadata
- Authors
- Xu, C. L., Cho, G. Y., Sengillo, J. D., Park, K. S., Mahajan, V. B., & Tsang, S. H.
- Venue
- Frontiers in Cell and Developmental Biology
- Publisher
- Frontiers Media SA
- Published
- 2018-05-23
- Volume
- 6
- ISSN
- 2296-634X
- PMC
- PMC5974543
- Cited by
- 19
- References
- 41
- Funders
- National Eye Institute (P30EY019007, R01EY018213, R01EY024698)
- National Cancer Institute (5P30CA013696)
- New York State Department of Health - Wadsworth Center (C029572)
- Foundation Fighting Blindness (C-NY05-0705-0312)
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- The paper
- Xu, C. L., Cho, G. Y., Sengillo, J. D., Park, K. S., Mahajan, V. B., & Tsang, S. H. (2018). Translation of CRISPR Genome Surgery to the Bedside for Retinal Diseases. Frontiers in Cell and Developmental Biology. https://doi.org/10.3389/fcell.2018.00046
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Bibliographic data: Semantic Scholar, OpenAlex, Crossref.
Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
