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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: kosofsky b. 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: kosofsky b. 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.
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: kosofsky b. 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.
Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2.
Sondhi D, Kaminsky SM, Hackett NR, Pagovich OE, Rosenberg JB, De BP, Chen A, Van de Graaf B, Mezey JG, Mammen GW, Mancenido D, Xu F, Kosofsky B, Yohay K, Worgall S, Kaner RJ, Souwedaine M, Greenwald BM, Kaplitt M, Dyke JP, Ballon DJ, Heier LA, Kiss S, Crystal RG. Sondhi D, et al. Among authors: kosofsky b. Sci Transl Med. 2020 Dec 2;12(572):eabb5413. doi: 10.1126/scitranslmed.abb5413. Sci Transl Med. 2020. PMID: 33268510 Free PMC article. Clinical Trial.
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: kosofsky b. Neurology. 2007 Aug 7;69(6):521-35. doi: 10.1212/01.wnl.0000267885.47092.40. Neurology. 2007. PMID: 17679671
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: kosofsky b. 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.
Brain Region-Specific Degeneration with Disease Progression in Late Infantile Neuronal Ceroid Lipofuscinosis (CLN2 Disease).
Dyke JP, Sondhi D, Voss HU, Yohay K, Hollmann C, Mancenido D, Kaminsky SM, Heier LA, Rudser KD, Kosofsky B, Casey BJ, Crystal RG, Ballon D. Dyke JP, et al. Among authors: kosofsky b. AJNR Am J Neuroradiol. 2016 Jun;37(6):1160-9. doi: 10.3174/ajnr.A4669. Epub 2016 Jan 28. AJNR Am J Neuroradiol. 2016. PMID: 26822727 Free PMC article.
112 results