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Clinical protocol. Administration of a replication-deficient adeno-associated virus gene transfer vector expressing the human CLN2 cDNA to the brain of children with late infantile neuronal ceroid lipofuscinosis.
Crystal RG, Sondhi D, Hackett NR, Kaminsky SM, Worgall S, Stieg P, Souweidane M, Hosain S, Heier L, Ballon D, Dinner M, Wisniewski K, Kaplitt M, Greenwald BM, Howell JD, Strybing K, Dyke J, Voss H. Crystal RG, et al. Among authors: worgall s. Hum Gene Ther. 2004 Nov;15(11):1131-54. doi: 10.1089/hum.2004.15.1131. Hum Gene Ther. 2004. PMID: 15610613 Clinical Trial.
Modification of the genetic program of human alveolar macrophages by adenovirus vectors in vitro is feasible but inefficient, limited in part by the low level of expression of the coxsackie/adenovirus receptor.
Kaner RJ, Worgall S, Leopold PL, Stolze E, Milano E, Hidaka C, Ramalingam R, Hackett NR, Singh R, Bergelson J, Finberg R, Falck-Pedersen E, Crystal RG. Kaner RJ, et al. Among authors: worgall s. Am J Respir Cell Mol Biol. 1999 Mar;20(3):361-70. doi: 10.1165/ajrcmb.20.3.3398. Am J Respir Cell Mol Biol. 1999. PMID: 10030833
115 results