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Page 1
Genetic defects in muscular dystrophy.
Chandrasekharan K, Martin PT. Chandrasekharan K, et al. Methods Enzymol. 2010;479:291-322. doi: 10.1016/S0076-6879(10)79017-0. Methods Enzymol. 2010. PMID: 20816173 Review.
Emerging drugs for Duchenne muscular dystrophy.
Malik V, Rodino-Klapac LR, Mendell JR. Malik V, et al. Expert Opin Emerg Drugs. 2012 Jun;17(2):261-77. doi: 10.1517/14728214.2012.691965. Expert Opin Emerg Drugs. 2012. PMID: 22632414 Free PMC article. Review.
Gene therapy for muscular dystrophy: lessons learned and path forward.
Mendell JR, Rodino-Klapac L, Sahenk Z, Malik V, Kaspar BK, Walker CM, Clark KR. Mendell JR, et al. Neurosci Lett. 2012 Oct 11;527(2):90-9. doi: 10.1016/j.neulet.2012.04.078. Epub 2012 May 17. Neurosci Lett. 2012. PMID: 22609847 Free PMC article. Review.
Vascular delivery of rAAVrh74.MCK.GALGT2 to the gastrocnemius muscle of the rhesus macaque stimulates the expression of dystrophin and laminin α2 surrogates.
Chicoine LG, Rodino-Klapac LR, Shao G, Xu R, Bremer WG, Camboni M, Golden B, Montgomery CL, Shontz K, Heller KN, Griffin DA, Lewis S, Coley BD, Walker CM, Clark KR, Sahenk Z, Mendell JR, Martin PT. Chicoine LG, et al. Mol Ther. 2014 Apr;22(4):713-24. doi: 10.1038/mt.2013.246. Epub 2013 Oct 22. Mol Ther. 2014. PMID: 24145553 Free PMC article.
Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D.
Mendell JR, Rodino-Klapac LR, Rosales XQ, Coley BD, Galloway G, Lewis S, Malik V, Shilling C, Byrne BJ, Conlon T, Campbell KJ, Bremer WG, Taylor LE, Flanigan KM, Gastier-Foster JM, Astbury C, Kota J, Sahenk Z, Walker CM, Clark KR. Mendell JR, et al. Ann Neurol. 2010 Nov;68(5):629-38. doi: 10.1002/ana.22251. Ann Neurol. 2010. PMID: 21031578 Free PMC article. Clinical Trial.
Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery.
Chicoine LG, Montgomery CL, Bremer WG, Shontz KM, Griffin DA, Heller KN, Lewis S, Malik V, Grose WE, Shilling CJ, Campbell KJ, Preston TJ, Coley BD, Martin PT, Walker CM, Clark KR, Sahenk Z, Mendell JR, Rodino-Klapac LR. Chicoine LG, et al. Mol Ther. 2014 Feb;22(2):338-347. doi: 10.1038/mt.2013.244. Epub 2013 Oct 23. Mol Ther. 2014. PMID: 24196577 Free PMC article.
An Isolated Limb Infusion Method Allows for Broad Distribution of rAAVrh74.MCK.GALGT2 to Leg Skeletal Muscles in the Rhesus Macaque.
Xu R, Jia Y, Zygmunt DA, Cramer ML, Crowe KE, Shao G, Maki AE, Guggenheim HN, Hood BC, Griffin DA, Peterson E, Bolon B, Cheatham JP, Cheatham SL, Flanigan KM, Rodino-Klapac LR, Chicoine LG, Martin PT. Xu R, et al. Mol Ther Methods Clin Dev. 2018 Jul 14;10:89-104. doi: 10.1016/j.omtm.2018.06.002. eCollection 2018 Sep 21. Mol Ther Methods Clin Dev. 2018. PMID: 30073180 Free PMC article.