Department of Radiology
Found 12 results
Filters: Keyword is Neuronal Ceroid-Lipofuscinoses  [Clear All Filters]
2020
Sondhi D, Kaminsky SM, Hackett NR, Pagovich OE, Rosenberg JB, De BP, Chen A, Van de Graaf B, Mezey JG, Mammen GW et al..  2020.  Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing .. Sci Transl Med. 12(572)
2018
Schulz A, Ajayi T, Specchio N, Reyes Ede Los, Gissen P, Ballon D, Dyke JP, Cahan H, Slasor P, Jacoby D et al..  2018.  Study of Intraventricular Cerliponase Alfa for CLN2 Disease.. N Engl J Med. 378(20):1898-1907.
Sindelar M, Dyke JP, Deeb RS, Sondhi D, Kaminsky SM, Kosofsky BE, Ballon DJ, Crystal RG, Gross SS.  2018.  Untargeted Metabolite Profiling of Cerebrospinal Fluid Uncovers Biomarkers for Severity of Late Infantile Neuronal Ceroid Lipofuscinosis (CLN2, Batten Disease).. Sci Rep. 8(1):15229.
2016
Dyke JP, Sondhi D, Voss HU, Yohay K, Hollmann C, Mancenido D, Kaminsky SM, Heier LA, Rudser KD, Kosofsky B et al..  2016.  Brain Region-Specific Degeneration with Disease Progression in Late Infantile Neuronal Ceroid Lipofuscinosis (CLN2 Disease).. AJNR Am J Neuroradiol. 37(6):1160-9.
2013
Dyke JP, Sondhi D, Voss HU, Shungu DC, Mao X, Yohay K, Worgall S, Hackett NR, Hollmann C, Yeotsas ME et al..  2013.  Assessment of disease severity in late infantile neuronal ceroid lipofuscinosis using multiparametric MR imaging.. AJNR Am J Neuroradiol. 34(4):884-9.
2012
Sondhi D, Johnson L, Purpura K, Monette S, Souweidane MM, Kaplitt MG, Kosofsky B, Yohay K, Ballon D, Dyke J et al..  2012.  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.. Hum Gene Ther Methods. 23(5):324-35.
2011
Pike LS, Tannous BA, Deliolanis NC, Hsich G, Morse D, Tung C-H, Sena-Esteves M, Breakefield XO.  2011.  Imaging gene delivery in a mouse model of congenital neuronal ceroid lipofuscinosis.. Gene Ther. 18(12):1173-8.
2008
Worgall S, Sondhi D, Hackett NR, Kosofsky B, Kekatpure MV, Neyzi N, Dyke JP, Ballon D, Heier L, Greenwald BM et al..  2008.  Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.. Hum Gene Ther. 19(5):463-74.
2007
Dyke JP, Voss HU, Sondhi D, Hackett NR, Worgall S, Heier LA, Kosofsky BE, Uluğ AM, Shungu DC, Mao X et al..  2007.  Assessing disease severity in late infantile neuronal ceroid lipofuscinosis using quantitative MR diffusion-weighted imaging.. AJNR Am J Neuroradiol. 28(7):1232-6.
Worgall S, Kekatpure MV, Heier L, Ballon D, Dyke JP, Shungu D, Mao X, Kosofsky B, Kaplitt MG, Souweidane MM et al..  2007.  Neurological deterioration in late infantile neuronal ceroid lipofuscinosis.. Neurology. 69(6):521-35.
2005
Arkin LM, Sondhi D, Worgall S, Suh LHyon K, Hackett NR, Kaminsky SM, Hosain SA, Souweidane MM, Kaplitt MG, Dyke JP et al..  2005.  Confronting the issues of therapeutic misconception, enrollment decisions, and personal motives in genetic medicine-based clinical research studies for fatal disorders.. Hum Gene Ther. 16(9):1028-36.
2004
Crystal RG, Sondhi D, Hackett NR, Kaminsky SM, Worgall S, Stieg P, Souweidane M, Hosain S, Heier L, Ballon D et al..  2004.  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.. Hum Gene Ther. 15(11):1131-54.