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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: ballon d. Hum Gene Ther. 2004 Nov;15(11):1131-54. doi: 10.1089/hum.2004.15.1131. Hum Gene Ther. 2004. PMID: 15610613 Clinical Trial.
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: ballon d. Hum Gene Ther. 2008 May;19(5):463-74. doi: 10.1089/hum.2008.022. Hum Gene Ther. 2008. PMID: 18473686 Clinical Trial.
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: ballon d. 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.
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: ballon dj. Hum Gene Ther. 2005 Sep;16(9):1028-36. doi: 10.1089/hum.2005.16.1028. Hum Gene Ther. 2005. PMID: 16149901 Review.
AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease.
Rosenberg JB, Kaplitt MG, De BP, Chen A, Flagiello T, Salami C, Pey E, Zhao L, Ricart Arbona RJ, Monette S, Dyke JP, Ballon DJ, Kaminsky SM, Sondhi D, Petsko GA, Paul SM, Crystal RG. Rosenberg JB, et al. Among authors: ballon dj. Hum Gene Ther Clin Dev. 2018 Mar;29(1):24-47. doi: 10.1089/humc.2017.231. Epub 2018 Mar 13. Hum Gene Ther Clin Dev. 2018. PMID: 29409358 Free PMC article.
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: ballon d. Neurology. 2007 Aug 7;69(6):521-35. doi: 10.1212/01.wnl.0000267885.47092.40. Neurology. 2007. PMID: 17679671
113 results