The DOI matches its Crossref record; 3 of the 3 references it cites were found in a registry or an index.
Rescue of Dystrophic Muscle Through U7 snRNA-Mediated Exon Skipping
Abstract
Most mutations in the dystrophin gene create a frameshift or a stop in the mRNA and are associated with severe Duchenne muscular dystrophy. Exon skipping that naturally occurs at low frequency sometimes eliminates the mutation and leads to the production of a rescued protein. We have achieved persistent exon skipping that removes the mutated exon on the dystrophin messenger mRNA of the mdx mouse, by a single administration of an AAV vector expressing antisense sequences linked to a modified U7 small nuclear RNA. We report the sustained production of functional dystrophin at physiological levels in entire groups of muscles and the correction of the muscular dystrophy.
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Cites
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
Manno, C. S., Chew, A. J., Hutchison, S., Larson, P. J., Herzog, R. W., Arruda, V. R., Tai, S. J., Ragni, M. V., Thompson, A., Ozelo, M., et al. (2003) · Blood · doi:10.1182/blood-2002-10-3296 · Cited by 749
Systemic delivery of genes to striated muscles using adeno-associated viral vectors
Gregorevic, P., Blankinship, M. J., Allen, J. M., Crawford, R. W., Meuse, L., Miller, D. G., Russell, D. W., & Chamberlain, J. S. (2004) · Nature Medicine · doi:10.1038/nm1085 · Cited by 620
Chao, H., Liu, Y., Rabinowitz, J., Li, C., Samulski, R. J., & Walsh, C. E. (2000) · Molecular Therapy · doi:10.1006/mthe.2000.0219 · Cited by 366
Cited by
Duan, D., Goemans, N., Takeda, S., Mercuri, E., & Aartsma-Rus, A. (2021) · Nature Reviews Disease Primers · doi:10.1038/s41572-021-00248-3 · Cited by 1,312
Lombardo, A., Genovese, P., Beausejour, C. M., Colleoni, S., Lee, Y.-L., Kim, K. A., Ando, D., Urnov, F. D., Galli, C., Gregory, P. D., et al. (2007) · Nature Biotechnology · doi:10.1038/nbt1353 · Cited by 845
Therapeutic targeting of splicing in cancer
Lee, S. C.-W., & Abdel-Wahab, O. (2016) · Nature Medicine · doi:10.1038/nm.4165 · Cited by 561
Tuffery-Giraud, S., Béroud, C., Leturcq, F., Yaou, R. B., Hamroun, D., Michel-Calemard, L., Moizard, M.-P., Bernard, R., Cossée, M., Boisseau, P., et al. (2009) · Human Mutation · doi:10.1002/humu.20976 · Cited by 377
Self-citationMingozzi, F., Meulenberg, J. J., Hui, D. J., Basner-Tschakarjan, E., Hasbrouck, N. C., Edmonson, S. A., Hutnick, N. A., Betts, M. R., Kastelein, J. J., Stroes, E. S., et al. (2009) · Blood · doi:10.1182/blood-2008-07-167510 · Cited by 295
Metadata
- Authors
- Goyenvalle, A., Vulin, A., Fougerousse, F., Leturcq, F., Kaplan, J.-C., Garcia, L., & Danos, O.
- Venue
- Science
- Published
- 2004-12-03
- Volume
- 306
- Issue
- 5702
- Pages
- 1796-1799
- ISSN
- 0036-8075 (ISSN-L)
- 1095-9203
- OpenAlex
- W2090849645
- Cited by
- 506
- References
- 3
- FWCI
- 12.57
- Type
- journal article
- Language
- en
- Open access
- closed
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- Goyenvalle, A., Vulin, A., Fougerousse, F., Leturcq, F., Kaplan, J.-C., Garcia, L., & Danos, O. (2004). Rescue of Dystrophic Muscle Through U7 snRNA-Mediated Exon Skipping. Science. https://doi.org/10.1126/science.1104297
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
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