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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.
Confronting the issues of therapeutic misconception, enrollment decisions, and personal motives in genetic medicine-based clinical research studies for fatal disorders.
Arkin LM, Sondhi D, Worgall S, Suh LH, Hackett NR, Kaminsky SM, Hosain SA, Souweidane MM, Kaplitt MG, Dyke JP, Heier LA, Ballon DJ, Shungu DC, Wisniewski KE, Greenwald BM, Hollmann C, Crystal RG. Arkin LM, et al. Among authors: souweidane mm. Hum Gene Ther. 2005 Sep;16(9):1028-36. doi: 10.1089/hum.2005.16.1028. Hum Gene Ther. 2005. PMID: 16149901 Review.
Neurological deterioration in late infantile neuronal ceroid lipofuscinosis.
Worgall S, Kekatpure MV, Heier L, Ballon D, Dyke JP, Shungu D, Mao X, Kosofsky B, Kaplitt MG, Souweidane MM, Sondhi D, Hackett NR, Hollmann C, Crystal RG. Worgall S, et al. Among authors: souweidane mm. Neurology. 2007 Aug 7;69(6):521-35. doi: 10.1212/01.wnl.0000267885.47092.40. Neurology. 2007. PMID: 17679671
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.
Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.
Worgall S, Sondhi D, Hackett NR, Kosofsky B, Kekatpure MV, Neyzi N, Dyke JP, Ballon D, Heier L, Greenwald BM, Christos P, Mazumdar M, Souweidane MM, Kaplitt MG, Crystal RG. Worgall S, et al. Among authors: souweidane mm. Hum Gene Ther. 2008 May;19(5):463-74. doi: 10.1089/hum.2008.022. Hum Gene Ther. 2008. PMID: 18473686 Clinical Trial.
224 results