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408 results

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Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy.
Malik V, Rodino-Klapac LR, Viollet L, Wall C, King W, Al-Dahhak R, Lewis S, Shilling CJ, Kota J, Serrano-Munuera C, Hayes J, Mahan JD, Campbell KJ, Banwell B, Dasouki M, Watts V, Sivakumar K, Bien-Willner R, Flanigan KM, Sahenk Z, Barohn RJ, Walker CM, Mendell JR. Malik V, et al. Among authors: mendell jr. Ann Neurol. 2010 Jun;67(6):771-80. doi: 10.1002/ana.22024. Ann Neurol. 2010. PMID: 20517938 Clinical Trial.
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.
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.
Update on the treatment of Duchenne muscular dystrophy.
Rodino-Klapac LR, Mendell JR, Sahenk Z. Rodino-Klapac LR, et al. Among authors: mendell jr. Curr Neurol Neurosci Rep. 2013 Mar;13(3):332. doi: 10.1007/s11910-012-0332-1. Curr Neurol Neurosci Rep. 2013. PMID: 23328943 Free PMC article. Review.
Eteplirsen for the treatment of Duchenne muscular dystrophy.
Mendell JR, Rodino-Klapac LR, Sahenk Z, Roush K, Bird L, Lowes LP, Alfano L, Gomez AM, Lewis S, Kota J, Malik V, Shontz K, Walker CM, Flanigan KM, Corridore M, Kean JR, Allen HD, Shilling C, Melia KR, Sazani P, Saoud JB, Kaye EM; Eteplirsen Study Group. Mendell JR, et al. Ann Neurol. 2013 Nov;74(5):637-47. doi: 10.1002/ana.23982. Epub 2013 Sep 10. Ann Neurol. 2013. PMID: 23907995 Free article. Clinical Trial.
Gene therapy for muscular dystrophy: moving the field forward.
Al-Zaidy S, Rodino-Klapac L, Mendell JR. Al-Zaidy S, et al. Among authors: mendell jr. Pediatr Neurol. 2014 Nov;51(5):607-18. doi: 10.1016/j.pediatrneurol.2014.08.002. Epub 2014 Aug 7. Pediatr Neurol. 2014. PMID: 25439576 Free PMC article. Review.
Duchenne muscular dystrophy: CRISPR/Cas9 treatment.
Mendell JR, Rodino-Klapac LR. Mendell JR, et al. Cell Res. 2016 May;26(5):513-4. doi: 10.1038/cr.2016.28. Epub 2016 Mar 1. Cell Res. 2016. PMID: 26926391 Free PMC article.
Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion.
Mendell JR, Chicoine LG, Al-Zaidy SA, Sahenk Z, Lehman K, Lowes L, Miller N, Alfano L, Galliers B, Lewis S, Murrey D, Peterson E, Griffin DA, Church K, Cheatham S, Cheatham J, Hogan MJ, Rodino-Klapac LR. Mendell JR, et al. Hum Gene Ther. 2019 Jul;30(7):794-801. doi: 10.1089/hum.2019.006. Epub 2019 Apr 19. Hum Gene Ther. 2019. PMID: 30838895 Free PMC article.
408 results