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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. Hum Gene Ther. 2004 Nov;15(11):1131-54. doi: 10.1089/hum.2004.15.1131. Hum Gene Ther. 2004. PMID: 15610613 Clinical Trial.
Use of human neural tissue for the generation of progenitors.
Greenfield JP, Ayuso-Sacido A, Schwartz TH, Pannullo S, Souweidane M, Stieg PE, Boockvar JA. Greenfield JP, et al. Neurosurgery. 2008 Jan;62(1):21-37; discussion 27-30. doi: 10.1227/01.NEU.0000311059.87873.46. Neurosurgery. 2008. PMID: 18300889 Review.
224 results