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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: kaminsky sm. Hum Gene Ther. 2004 Nov;15(11):1131-54. doi: 10.1089/hum.2004.15.1131. Hum Gene Ther. 2004. PMID: 15610613 Clinical Trial.
AAV2-mediated CLN2 gene transfer to rodent and non-human primate brain results in long-term TPP-I expression compatible with therapy for LINCL.
Sondhi D, Peterson DA, Giannaris EL, Sanders CT, Mendez BS, De B, Rostkowski AB, Blanchard B, Bjugstad K, Sladek JR Jr, Redmond DE Jr, Leopold PL, Kaminsky SM, Hackett NR, Crystal RG. Sondhi D, et al. Among authors: kaminsky sm. Gene Ther. 2005 Nov;12(22):1618-32. doi: 10.1038/sj.gt.3302549. Gene Ther. 2005. PMID: 16052206
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: kaminsky sm. Hum Gene Ther. 2005 Sep;16(9):1028-36. doi: 10.1089/hum.2005.16.1028. Hum Gene Ther. 2005. PMID: 16149901 Review.
Safety of direct administration of AAV2(CU)hCLN2, a candidate treatment for the central nervous system manifestations of late infantile neuronal ceroid lipofuscinosis, to the brain of rats and nonhuman primates.
Hackett NR, Redmond DE, Sondhi D, Giannaris EL, Vassallo E, Stratton J, Qiu J, Kaminsky SM, Lesser ML, Fisch GS, Rouselle SD, Crystal RG. Hackett NR, et al. Among authors: kaminsky sm. Hum Gene Ther. 2005 Dec;16(12):1484-503. doi: 10.1089/hum.2005.16.1484. Hum Gene Ther. 2005. PMID: 16390279
Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis.
Passini MA, Dodge JC, Bu J, Yang W, Zhao Q, Sondhi D, Hackett NR, Kaminsky SM, Mao Q, Shihabuddin LS, Cheng SH, Sleat DE, Stewart GR, Davidson BL, Lobel P, Crystal RG. Passini MA, et al. Among authors: kaminsky sm. J Neurosci. 2006 Feb 1;26(5):1334-42. doi: 10.1523/JNEUROSCI.2676-05.2006. J Neurosci. 2006. PMID: 16452657 Free PMC article.
81 results