Phase I trial of adenoviral gene transfer in ornithine transcarbamylase deficiency † 731

被引:0
|
作者
Steven Raper
James M Wilson
Marc Yudkoff
Susan Magosin
Mark L Batshaw
机构
[1] Surgery,
[2] Pediatrics,undefined
[3] University of Pennsylvania School of Medicine,undefined
[4] Children's Seashore House,undefined
[5] Institute for Human Gene Therapy,undefined
[6] Children's Hospital of Philadelphia,undefined
关键词
D O I
10.1203/00006450-199804001-00752
中图分类号
学科分类号
摘要
引用
收藏
页码:127 / 127
相关论文
共 50 条
  • [1] Developing adenoviral-mediated in vivo gene therapy for ornithine transcarbamylase deficiency
    Raper, SE
    Wilson, JM
    Yudkoff, M
    Robinson, MB
    Ye, X
    Batshaw, ML
    [J]. JOURNAL OF INHERITED METABOLIC DISEASE, 1998, 21 : 119 - 137
  • [2] A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine Transcarbamylase deficiency
    Raper, SE
    Yudkoff, M
    Chirmule, N
    Gao, GP
    Nunes, F
    Haskal, ZJ
    Furth, EE
    Propert, KJ
    Robinson, MB
    Magosin, S
    Simoes, H
    Speicher, L
    Hughes, J
    Tazelaar, J
    Wivel, NA
    Wilson, JM
    Batshaw, ML
    [J]. HUMAN GENE THERAPY, 2002, 13 (01) : 163 - 175
  • [3] Gene therapy for ornithine transcarbamylase deficiency
    Kiwaki, K
    Matsuda, I
    [J]. ACTA PAEDIATRICA JAPONICA, 1996, 38 (02): : 189 - 192
  • [4] Lessons learned from the gene therapy trial for ornithine transcarbamylase deficiency
    Wilson, James M.
    [J]. MOLECULAR GENETICS AND METABOLISM, 2009, 96 (04) : 151 - 157
  • [5] Recombinant adenovirus gene transfer in adults with partial ornithine transcarbamylase deficiency (OTCD)
    Batshaw, ML
    Wilson, JM
    Raper, S
    [J]. HUMAN GENE THERAPY, 1999, 10 (14) : 2419 - 2437
  • [6] CORRECTION OF MOUSE ORNITHINE TRANSCARBAMYLASE DEFICIENCY BY GENE-TRANSFER INTO THE GERM LINE
    CAVARD, C
    GRIMBER, G
    DUBOIS, N
    CHASSE, JF
    BENNOUN, M
    MINETTHURIAUX, M
    KAMOUN, P
    BRIAND, P
    [J]. NUCLEIC ACIDS RESEARCH, 1988, 16 (05) : 2099 - 2110
  • [7] A novel mutation in ornithine transcarbamylase gene identified from a Chinese child with ornithine transcarbamylase deficiency
    Wu, Bo
    Wang, Yi-Zheng
    Cao, Li-Rong
    Zhang, Rui-Ping
    Fang, Yu-Lian
    Zhang, Yu-Qin
    Cai, Chun-Quan
    [J]. INTERNATIONAL JOURNAL OF CLINICAL AND EXPERIMENTAL MEDICINE, 2018, 11 (06): : 6344 - 6350
  • [8] Gene Mutation Analysis and Prenatal Diagnosis of the Ornithine Transcarbamylase (OTC) Gene in Two Families with Ornithine Transcarbamylase Deficiency
    Li, Sitao
    Cai, Yao
    Shi, Congcong
    Liu, Mengxian
    Liu, Bingqing
    Lin, Lin
    Xiao, Xin
    Hao, Hu
    [J]. MEDICAL SCIENCE MONITOR, 2018, 24 : 7431 - 7437
  • [9] n of 1 trial for an ornithine transcarbamylase deficiency carrier
    Hackett, A.
    Gillard, J.
    Wilcken, B.
    [J]. MOLECULAR GENETICS AND METABOLISM, 2008, 94 (02) : 157 - 161
  • [10] Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer
    Raper, SE
    Chirmule, N
    Lee, FS
    Wivel, NA
    Bagg, A
    Gao, GP
    Wilson, JM
    Batshaw, ML
    [J]. MOLECULAR GENETICS AND METABOLISM, 2003, 80 (1-2) : 148 - 158