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7,217 results

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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. Among authors: lewis s. 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.
Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins.
Mendell JR, Rodino-Klapac LR, Rosales-Quintero X, Kota J, Coley BD, Galloway G, Craenen JM, Lewis S, Malik V, Shilling C, Byrne BJ, Conlon T, Campbell KJ, Bremer WG, Viollet L, Walker CM, Sahenk Z, Clark KR. Mendell JR, et al. Among authors: lewis s. Ann Neurol. 2009 Sep;66(3):290-7. doi: 10.1002/ana.21732. Ann Neurol. 2009. PMID: 19798725 Free PMC article. Clinical Trial.
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: lewis s. Ann Neurol. 2010 Jun;67(6):771-80. doi: 10.1002/ana.22024. Ann Neurol. 2010. PMID: 20517938 Clinical Trial.
Novel diagnostic features of dysferlinopathies.
Rosales XQ, Gastier-Foster JM, Lewis S, Vinod M, Thrush DL, Astbury C, Pyatt R, Reshmi S, Sahenk Z, Mendell JR. Rosales XQ, et al. Among authors: lewis s. Muscle Nerve. 2010 Jul;42(1):14-21. doi: 10.1002/mus.21650. Muscle Nerve. 2010. PMID: 20544924 Free PMC article.
Dystrophin immunity in Duchenne's muscular dystrophy.
Mendell JR, Campbell K, Rodino-Klapac L, Sahenk Z, Shilling C, Lewis S, Bowles D, Gray S, Li C, Galloway G, Malik V, Coley B, Clark KR, Li J, Xiao X, Samulski J, McPhee SW, Samulski RJ, Walker CM. Mendell JR, et al. Among authors: lewis s. N Engl J Med. 2010 Oct 7;363(15):1429-37. doi: 10.1056/NEJMoa1000228. N Engl J Med. 2010. PMID: 20925545 Free PMC article. 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. Among authors: lewis s. Ann Neurol. 2010 Nov;68(5):629-38. doi: 10.1002/ana.22251. Ann Neurol. 2010. PMID: 21031578 Free PMC article. Clinical Trial.
Impaired regeneration in LGMD2A supported by increased PAX7-positive satellite cell content and muscle-specific microrna dysregulation.
Rosales XQ, Malik V, Sneh A, Chen L, Lewis S, Kota J, Gastier-Foster JM, Astbury C, Pyatt R, Reshmi S, Rodino-Klapac LR, Clark KR, Mendell JR, Sahenk Z. Rosales XQ, et al. Among authors: lewis s. Muscle Nerve. 2013 May;47(5):731-9. doi: 10.1002/mus.23669. Epub 2013 Mar 29. Muscle Nerve. 2013. PMID: 23553538 Free PMC article.
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. Among authors: lewis s. 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.
7,217 results