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912 results

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Page 1
Long-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis.
Sondhi D, Johnson L, Purpura K, Monette S, Souweidane MM, Kaplitt MG, Kosofsky B, Yohay K, Ballon D, Dyke J, Kaminksy SM, Hackett NR, Crystal RG. Sondhi D, et al. Among authors: crystal rg. Hum Gene Ther Methods. 2012 Oct;23(5):324-35. doi: 10.1089/hgtb.2012.120. Epub 2012 Nov 6. Hum Gene Ther Methods. 2012. PMID: 23131032 Free PMC article.
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: crystal rg. 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: crystal rg. Hum Gene Ther. 2005 Dec;16(12):1484-503. doi: 10.1089/hum.2005.16.1484. Hum Gene Ther. 2005. PMID: 16390279
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: crystal rg. Hum Gene Ther. 2008 May;19(5):463-74. doi: 10.1089/hum.2008.022. Hum Gene Ther. 2008. PMID: 18473686 Clinical Trial.
912 results