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Page 1
Spectrum of ocular manifestations in CLN2-associated batten (Jansky-Bielschowsky) disease correlate with advancing age and deteriorating neurological function.
Orlin A, Sondhi D, Witmer MT, Wessel MM, Mezey JG, Kaminsky SM, Hackett NR, Yohay K, Kosofsky B, Souweidane MM, Kaplitt MG, D'Amico DJ, Crystal RG, Kiss S. Orlin A, et al. Among authors: souweidane mm. PLoS One. 2013 Aug 28;8(8):e73128. doi: 10.1371/journal.pone.0073128. eCollection 2013. PLoS One. 2013. PMID: 24015292 Free PMC article. Clinical Trial.
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
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.
Assessment of disease severity in late infantile neuronal ceroid lipofuscinosis using multiparametric MR imaging.
Dyke JP, Sondhi D, Voss HU, Shungu DC, Mao X, Yohay K, Worgall S, Hackett NR, Hollmann C, Yeotsas ME, Jeong AL, Van de Graaf B, Cao I, Kaminsky SM, Heier LA, Rudser KD, Souweidane MM, Kaplitt MG, Kosofsky B, Crystal RG, Ballon D. Dyke JP, et al. Among authors: souweidane mm. AJNR Am J Neuroradiol. 2013 Apr;34(4):884-9. doi: 10.3174/ajnr.A3297. Epub 2012 Oct 4. AJNR Am J Neuroradiol. 2013. PMID: 23042927 Free PMC article.
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: souweidane mm. 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.
Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates.
Zerah M, Piguet F, Colle MA, Raoul S, Deschamps JY, Deniaud J, Gautier B, Toulgoat F, Bieche I, Laurendeau I, Sondhi D, Souweidane MM, Cartier-Lacave N, Moullier P, Crystal RG, Roujeau T, Sevin C, Aubourg P. Zerah M, et al. Among authors: souweidane mm. Hum Gene Ther Clin Dev. 2015 Jun;26(2):113-24. doi: 10.1089/humc.2014.139. Epub 2015 Apr 28. Hum Gene Ther Clin Dev. 2015. PMID: 25758611 Clinical Trial.
225 results