Long-Term Correction of Glycogen Storage Disease Type III by AAV-Mediated Gene Therapy Using a Dual Promoter to Express Bacterial Pullulanase

被引:0
|
作者
Lim, Jeong-A [1 ]
Choi, Su Jin [1 ]
Kishnani, Priya S. [1 ]
Sun, Baodong [1 ]
机构
[1] Duke Univ, Durham, NC USA
关键词
D O I
暂无
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
246
引用
收藏
页码:119 / 120
页数:2
相关论文
共 50 条
  • [21] AAV-Mediated Gene Therapy Using a Novel Dual Promoter Prevents Pullulanase-Induced Cytotoxic T Lymphocyte Response and Corrects Major Affected Tissues in GSD IIIa Mice
    Lim, Jeong-A
    Kishnani, Priya S.
    Sun, Baodong
    MOLECULAR THERAPY, 2021, 29 (04) : 235 - 236
  • [22] Long-term complications of glycogen storage disease Type IA in the dog model treated with gene replacement therapy
    Brooks, Elizabeth D.
    Landau, Dustin J.
    Asfaw, Yohannes
    Koeberl, Dwight D.
    MOLECULAR GENETICS AND METABOLISM, 2015, 114 (03) : 312 - 312
  • [23] Long-term complications of glycogen storage disease type Ia in the canine model treated with gene replacement therapy
    Brooks, Elizabeth D.
    Landau, Dustin J.
    Everitt, Jeffrey I.
    Brown, Talmage T.
    Grady, Kylie M.
    Waskowicz, Lauren
    Bass, Cameron R.
    D'Angelo, John
    Asfaw, Yohannes G.
    Williams, Kyha
    Kishnani, Priya S.
    Koeberl, Dwight D.
    JOURNAL OF INHERITED METABOLIC DISEASE, 2018, 41 (06) : 965 - 976
  • [24] Long-Term Complications of Glycogen Storage Disease Type IA in the Dog Model Treated With Gene Replacement Therapy
    Brooks, Elizabeth D.
    Landau, Dustin J.
    Brown, Talmage T.
    Asfaw, Yohannes G.
    Koeberl, Dwight D.
    MOLECULAR THERAPY, 2015, 23 : S279 - S279
  • [25] LONG-TERM CORNSTARCH THERAPY IN GLYCOGEN-STORAGE-DISEASE TYPE-I, TYPE-IB AND TYPE-III
    GATTI, R
    LAMEDICA, G
    DIROCCO, M
    MASSOCCO, D
    MARCHESE, N
    BORRONE, C
    JOURNAL OF INHERITED METABOLIC DISEASE, 1986, 9 : 280 - 283
  • [26] A novel glioblastoma cancer gene therapy using AAV-mediated long-term expression of human TERT C-terminal polypeptide
    S S M Ng
    Y Gao
    D H W Chau
    G H Y Li
    L H Lai
    P T Huang
    C F Huang
    J J Huang
    Y C Chen
    H F Kung
    M C M Lin
    Cancer Gene Therapy, 2007, 14 : 561 - 572
  • [27] A novel glioblastoma cancer gene therapy using AAV-mediated long-term expression of human TERT C-terminal polypeptide
    Ng, S. S. M.
    Gao, Y.
    Chau, D. H. W.
    Li, G. H. Y.
    Lai, L. H.
    Huang, P. T.
    Huang, C. F.
    Huang, J. J.
    Chen, Y. C.
    Kung, H. F.
    Lin, M. C. M.
    CANCER GENE THERAPY, 2007, 14 (06) : 561 - 572
  • [28] Long-term amelioration of an early-onset familial atrial fibrillation model with AAV-mediated in vivo gene therapy
    Hu, Handan
    Wang, Liren
    Li, Huiying
    Li, Hailing
    Chen, Xi
    Peng, Wenhui
    Wang, Jun
    Zhao, Yongxiang
    Liu, Mingyao
    Li, Dali
    FUNDAMENTAL RESEARCH, 2022, 2 (06): : 829 - 835
  • [29] Long-term correction of murine glycogen storage disease type Ia by recombinant adeno-associated virus-1-mediated gene transfer
    A Ghosh
    M Allamarvdasht
    C-J Pan
    M-S Sun
    B C Mansfield
    B J Byrne
    J Y Chou
    Gene Therapy, 2006, 13 : 321 - 329
  • [30] Long-term correction of murine glycogen storage disease type Ia by recombinant adeno-associated virus-1-mediated gene transfer
    Ghosh, A
    Allamarvdasht, M
    Pan, CJ
    Sun, MS
    Mansfield, BC
    Byrne, BJ
    Chou, JY
    GENE THERAPY, 2006, 13 (04) : 321 - 329