The DOI matches its Crossref record; 10 of the 10 references it cites were found in a registry or an index.
CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells
Open accessAbstract
A mouse model with a common human DMD mutation was created and corrected by CRISPR-Cas9 gene editing.
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Cites
A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity
Jinek, M., Chylinski, K., Fonfara, I., Hauer, M., Doudna, J. A., & Charpentier, E. (2012) · Science · doi:10.1126/science.1225829 · Cited by 17,116
Genome engineering using the CRISPR-Cas9 system
Ran, F. A., Hsu, P. D., Wright, J., Agarwala, V., Scott, D. A., & Zhang, F. (2013) · Nature Protocols · doi:10.1038/nprot.2013.143 · Cited by 11,133
Dystrophin: the protein product of the Duchenne muscular dystrophy locus
Hoffman, E. P., Brown, R. H., & Kunkel, L. M. (1987) · Cell · doi:10.1016/0092-8674(87)90579-4 · Cited by 4,693
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
Mendell, J. R., Al-Zaidy, S., Shell, R., Arnold, W. D., Rodino-Klapac, L. R., Prior, T. W., Lowes, L., Alfano, L., Berry, K., Church, K., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1706198 · Cited by 2,381
Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection
Zincarelli, C., Soltys, S., Rengo, G., & Rabinowitz, J. E. (2008) · Molecular Therapy · doi:10.1038/mt.2008.76 · Cited by 1,490
Structures of Cas9 endonucleases reveal RNA-mediated conformational activation
Jinek, M., Jiang, F., Taylor, D. W., Sternberg, S. H., Kaya, E., Ma, E., Anders, C., Hauer, M., Zhou, K., Lin, S., et al. (2014) · Science · doi:10.1126/science.1247997 · Cited by 1,230
In vivo gene editing in dystrophic mouse muscle and muscle stem cells
Tabebordbar, M., Zhu, K., Cheng, J. K. W., Chew, W. L., Widrick, J. J., Yan, W. X., Maesner, C., Wu, E. Y., Xiao, R., Ran, F. A., et al. (2016) · Science · doi:10.1126/science.aad5177 · Cited by 1,046
AAV vector integration sites in mouse hepatocellular carcinoma
Donsante, A., Miller, D. G., Li, Y., Vogler, C., Brunt, E. M., Russell, D. W., & Sands, M. S. (2007) · Science · doi:10.1126/science.1142658 · Cited by 662
Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy
Amoasii, L., Hildyard, J. C. W., Li, H., Sanchez-Ortiz, E., Mireault, A., Caballero, D., Harron, R., Stathopoulou, T.-R., Massey, C., Shelton, J. M., et al. (2018) · Science · doi:10.1126/science.aau1549 · Cited by 546
Self-citationOusterout, D. G., Kabadi, A. M., Thakore, P. I., Majoros, W. H., Reddy, T. E., & Gersbach, C. A. (2015) · Nature Communications · doi:10.1038/ncomms7244 · Cited by 470
Cited by
CRISPR-Cas9: A Preclinical and Clinical Perspective for the Treatment of Human Diseases
Sharma, G., Sharma, A. R., Bhattacharya, M., Lee, S.-S., & Chakraborty, C. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2020.09.028 · Cited by 283
Delivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and challenges
Taha, E. A., Lee, J., & Hotta, A. (2022) · Journal of Controlled Release · doi:10.1016/j.jconrel.2022.01.013 · Cited by 259
Lipid nanoparticle-mediated drug delivery to the brain
Khare, P., Edgecomb, S. X., Hamadani, C. M., Tanner, E. E. L., & S Manickam, D. (2023) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2023.114861 · Cited by 187
Gonzalez, T. J., Simon, K. E., Blondel, L. O., Fanous, M. M., Roger, A. L., Maysonet, M. S., Devlin, G. W., Smith, T. J., Oh, D. K., Havlik, L. P., et al. (2022) · Nature Communications · doi:10.1038/s41467-022-33745-4 · Cited by 85
Innovative Therapeutic Approaches for Duchenne Muscular Dystrophy
Fortunato, F., Rossi, R., Falzarano, M. S., & Ferlini, A. (2021) · Journal of Clinical Medicine · doi:10.3390/jcm10040820 · Cited by 64
Metadata
- Authors
- Min, Y.-L., Li, H., Rodriguez-Caycedo, C., Mireault, A. A., Huang, J., Shelton, J. M., McAnally, J. R., Amoasii, L., Mammen, P. P. A., Bassel-Duby, R., & Olson, E. N.
- Venue
- Science Advances
- Published
- 2019-03
- Volume
- 5
- Issue
- 3
- Pages
- eaav4324-eaav4324
- ISSN
- 2375-2548 (ISSN-L)
- OpenAlex
- W2918808095
- Cited by
- 251
- References
- 10
- FWCI
- 11.24
- Type
- journal article
- Language
- en
- Open access
- gold
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- CoChat. (2026). Citation check: CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells. https://app.cochat.ai/papers/10.1126/sciadv.aav4324
- The paper
- Min, Y.-L., Li, H., Rodriguez-Caycedo, C., Mireault, A. A., Huang, J., Shelton, J. M., McAnally, J. R., Amoasii, L., Mammen, P. P. A., Bassel-Duby, R., & Olson, E. N. (2019). CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells. Science Advances. https://doi.org/10.1126/sciadv.aav4324
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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.
