Creation of Dystrophin Expressing Chimeric Cells of Myoblast Origin as a Novel Stem Cell Based Therapy for Duchenne Muscular Dystrophy
Author:
Funder
Office of the Vice Chancellor for Research, University of Illinois at Chicago
Publisher
Springer Science and Business Media LLC
Subject
Cancer Research,Cell Biology
Link
http://link.springer.com/article/10.1007/s12015-017-9792-7/fulltext.html
Reference49 articles.
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2. Kinali, M., Arechavala-Gomeza, V., Feng, L., et al. (2009). Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study. Lancet Neurol, 8, 918 – 28.
3. Nelson, C. E., Hakim, C. H., Ousterout, D. G., et al. (2016). In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy. Science, 351, 403–407.
4. Long, C., McAnally, J. R., Shelton, J. M., Mireault, A. A., Bassel-Duby, R., & Olson, E. N. (2014). Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA. Science, 345, 1184–1188.
5. Sienkiewicz, D., Kulak, W., Okurowska-Zawada, B., Paszko-Patej, G., & Kawnik, K. (2015). Duchenne muscular dystrophy: current cell therapies. Ther Adv Neurol Disord, 8, 166 – 77.
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