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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: conlon t. Ann Neurol. 2009 Sep;66(3):290-7. doi: 10.1002/ana.21732. Ann Neurol. 2009. PMID: 19798725 Free PMC article. Clinical Trial.
Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults.
Brantly ML, Spencer LT, Humphries M, Conlon TJ, Spencer CT, Poirier A, Garlington W, Baker D, Song S, Berns KI, Muzyczka N, Snyder RO, Byrne BJ, Flotte TR. Brantly ML, et al. Among authors: conlon tj. Hum Gene Ther. 2006 Dec;17(12):1177-86. doi: 10.1089/hum.2006.17.1177. Hum Gene Ther. 2006. PMID: 17115945 Clinical Trial.
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy.
Brantly ML, Chulay JD, Wang L, Mueller C, Humphries M, Spencer LT, Rouhani F, Conlon TJ, Calcedo R, Betts MR, Spencer C, Byrne BJ, Wilson JM, Flotte TR. Brantly ML, et al. Proc Natl Acad Sci U S A. 2009 Sep 22;106(38):16363-8. doi: 10.1073/pnas.0904514106. Epub 2009 Aug 12. Proc Natl Acad Sci U S A. 2009. PMID: 19706466 Free PMC article. Clinical Trial.
Adeno-associated virus-mediated correction of a canine model of glycogen storage disease type Ia.
Weinstein DA, Correia CE, Conlon T, Specht A, Verstegen J, Onclin-Verstegen K, Campbell-Thompson M, Dhaliwal G, Mirian L, Cossette H, Falk DJ, Germain S, Clement N, Porvasnik S, Fiske L, Struck M, Ramirez HE, Jordan J, Andrutis K, Chou JY, Byrne BJ, Mah CS. Weinstein DA, et al. Among authors: conlon t. Hum Gene Ther. 2010 Jul;21(7):903-10. doi: 10.1089/hum.2009.157. Hum Gene Ther. 2010. PMID: 20163245 Free PMC article.
208 results