HIGHLY EFFICACIOUS GENE THERAPY IN THE CANINE MODEL FOR GLYCOGEN STORAGE DISEASE TYPE IA

被引：0
|
作者：
Luo, Xiaoyan [1 ]
Demaster, Amanda [1 ]
Crane, Bayley [2 ]
Pinto, C. R. [2 ]
Brown, T. T. [2 ]
Kozink, D. M. [2 ]
Bird, A. [1 ]
Kishnani, P. S. [1 ]
Koeberl, D. D. [1 ]
机构：
[1] Duke Univ, Med Ctr, Dept Pediat, Div Med Genet, Durham, NC 27710 USA
[2] N Carolina State Univ, Coll Vet Med, Raleigh, NC USA
来源：
MOLECULAR GENETICS AND METABOLISM | 2010年 / 99卷 / 03期
关键词：
D O I：
暂无
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
引用
收藏
页码：221 / 222
页数：2
相关论文
共 50 条
  • [1] Efficacy of gene therapy in canine glycogen storage disease type IA.
    Pinto, C. R.
    Brown, T. R.
    Vaughn, V.
    Bird, A.
    Sun, B. D.
    Koeberl, D. D.
    JOURNAL OF VETERINARY INTERNAL MEDICINE, 2007, 21 (03) : 606 - 606
  • [2] Highly efficacious gene therapy in glycogen storage disease type Ia (GSD-Ia) with a double-stranded AAV vector.
    Koeberl, D. D.
    Sun, B.
    Pinto, C.
    Brown, T.
    Bird, A.
    Chen, Y. T.
    MOLECULAR GENETICS AND METABOLISM, 2007, 90 (03) : 255 - 256
  • [3] Genome Editing in a Canine Model for Glycogen Storage Disease Type Ia
    Arnson, Benjamin
    Kang, Hye Ri
    Brooks, Elizabeth
    Gheorghiu, Dorothy
    Li, Songtao
    Ilich, Ekaterina
    Cullen, Bryan
    Koeberl, Dwight D.
    MOLECULAR THERAPY, 2022, 30 (04) : 219 - 219
  • [4] 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
  • [5] Long-term safety and efficacy of AAV gene therapy in the canine model of glycogen storage disease type Ia
    Lee, Young Mok
    Conlon, Thomas J.
    Specht, Andrew
    Coleman, Kirsten E.
    Brown, Laurie M.
    Estrella, Ana M.
    Dambska, Monika
    Dahlberg, Kathryn R.
    Weinstein, David A.
    JOURNAL OF INHERITED METABOLIC DISEASE, 2018, 41 (06) : 977 - 984
  • [6] Canine model and genomic structural organization of glycogen storage disease type Ia (GSD Ia)
    Kishnani, PS
    Faulkner, E
    VanCamp, S
    Jackson, M
    Brown, T
    Boney, A
    Koeberl, D
    Chen, YT
    VETERINARY PATHOLOGY, 2001, 38 (01) : 83 - 91
  • [7] Pathogenesis of growth failure and partial reversal with gene therapy in murine and canine Glycogen Storage Disease type Ia
    Brooks, Elizabeth Drake
    Little, Dianne
    Arumugam, Ramamani
    Sun, Baodong
    Curtis, Sarah
    DeMaster, Amanda
    Maranzano, Michael
    Jackson, Mark W.
    Kishnani, Priya
    Freemark, Michael S.
    Koeberl, Dwight D.
    MOLECULAR GENETICS AND METABOLISM, 2013, 109 (02) : 161 - 170
  • [8] Bezafibrate Enhances AAV Gene Therapy in Glycogen Storage Disease Type Ia
    Kang, Hye Ri
    Waskowicz, Lauren
    Koeberl, Dwight D.
    MOLECULAR THERAPY, 2018, 26 (05) : 447 - 447
  • [9] Gene therapy with adeno-associated virus (AAV) vectors in canine glycogen storage disease, type Ia.
    Beaty, RM
    Jackson, M
    Kishnani, PS
    Faulkner, E
    VanCamp, S
    Brown, T
    Boney, A
    Chen, YT
    Koeberl, DD
    AMERICAN JOURNAL OF HUMAN GENETICS, 2000, 67 (04) : 432 - 432
  • [10] Long term treatment of glycogen storage disease type IA: Clinical summary of a canine model
    Drake, Elizabeth J.
    Demaster, Amanda
    Luo, Xiaoyan
    Curtis, Sarah
    Williams, Kyha D.
    Landau, Dustin J.
    Kozink, Daniel M.
    Bird, Andrew
    Crane, Bayley
    Sun, Francis
    Pinto, Carlos R.
    Brown, Talmage T.
    Kemper, Alex R.
    Koeberl, Dwight D.
    MOLECULAR GENETICS AND METABOLISM, 2012, 105 (03) : 314 - 314
12345