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Pompe disease gene therapy.
Byrne BJ, Falk DJ, Pacak CA, Nayak S, Herzog RW, Elder ME, Collins SW, Conlon TJ, Clement N, Cleaver BD, Cloutier DA, Porvasnik SL, Islam S, Elmallah MK, Martin A, Smith BK, Fuller DD, Lawson LA, Mah CS. Byrne BJ, et al. Among authors: clement n. Hum Mol Genet. 2011 Apr 15;20(R1):R61-8. doi: 10.1093/hmg/ddr174. Epub 2011 Apr 25. Hum Mol Genet. 2011. PMID: 21518733 Free PMC article. Review.
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: clement n. Hum Gene Ther. 2010 Jul;21(7):903-10. doi: 10.1089/hum.2009.157. Hum Gene Ther. 2010. PMID: 20163245 Free PMC article.
Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes.
Smith BK, Collins SW, Conlon TJ, Mah CS, Lawson LA, Martin AD, Fuller DD, Cleaver BD, Clément N, Phillips D, Islam S, Dobjia N, Byrne BJ. Smith BK, et al. Among authors: clement n. Hum Gene Ther. 2013 Jun;24(6):630-40. doi: 10.1089/hum.2012.250. Hum Gene Ther. 2013. PMID: 23570273 Free PMC article. Clinical Trial.
Preclinical potency and safety studies of an AAV2-mediated gene therapy vector for the treatment of MERTK associated retinitis pigmentosa.
Conlon TJ, Deng WT, Erger K, Cossette T, Pang JJ, Ryals R, Clément N, Cleaver B, McDoom I, Boye SE, Peden MC, Sherwood MB, Abernathy CR, Alkuraya FS, Boye SL, Hauswirth WW. Conlon TJ, et al. Among authors: clement n. Hum Gene Ther Clin Dev. 2013 Mar;24(1):23-8. doi: 10.1089/humc.2013.037. Epub 2013 Apr 3. Hum Gene Ther Clin Dev. 2013. PMID: 23692380 Free PMC article.
567 results