Letter to the Editors: Concerning BLong-term safety and efficacy of AAV gene therapy in the canine model of glycogen storage disease type Ia" by Lee et al Response

被引:1
|
作者
Lee, Youngmok [1 ]
Chou, Janice Y. [2 ]
Weinstein, David A. [1 ]
机构
[1] Univ Connecticut, Hlth Ctr, Farmington, CT 06030 USA
[2] Eunice Kennedy Shriver Natl Inst Child Hlth & Hum, Sect Cellular Differentiat, Bethesda, MD USA
关键词
PREVENTION; LIVER;
D O I
10.1007/s10545-018-0249-1
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
引用
收藏
页码:915 / 915
页数:1
相关论文
共 46 条
  • [21] Efficacy of helper-dependent adenovirus vector-mediated gene therapy in murine glycogen storage disease type Ia
    Koeberl, Dwight D.
    Sun, B.
    Bird, A.
    Chen, Y. T.
    Oka, K.
    Chan, L.
    MOLECULAR THERAPY, 2007, 15 (07) : 1253 - 1258
  • [22] Safety and Efficacy of DTX401, an AAV8-Mediated Liver-Directed Gene Therapy, in Adults With Glycogen Storage Disease Type I a (GSDIa)
    Weinstein, David A.
    Derks, Terry G.
    Rodriguez-Buritica, David F.
    Ahmad, Ayesha
    Couce, Maria-Luz
    Mitchell, John J.
    Riba-Wolman, Rebecca
    Mount, Malaya
    Sallago, Julieta Bonvin
    Ross, Katalin M.
    van Der Klauw, Melanie M.
    de Boer, Foekje
    van Der Schaaf, Caroline
    Saavedra, Heather
    Martinez-Olmos, Miguel
    Atanga, Elvis
    Hosseini, Asad
    Mitragotri, Deepali
    Crombez, Eric
    JOURNAL OF INHERITED METABOLIC DISEASE, 2025, 48 (02)
  • [23] Adeno-Associated Virus Gene Therapy Prevents Hepatocellular Adenoma in Murine Model of Glycogen Storage Disease Type Ia
    Wang, Lili
    HEPATOLOGY, 2012, 56 (05) : 1593 - 1595
  • [24] Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia
    Koeberl, D. D.
    Sun, B. D.
    Damodaran, T. V.
    Brown, T.
    Millington, D. S.
    Benjamin, D. J., Jr.
    Bird, A.
    Schneider, A.
    Hillman, S.
    Jackson, M.
    Beaty, R. M.
    Chen, Y. T.
    GENE THERAPY, 2006, 13 (17) : 1281 - 1289
  • [25] Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia
    D D Koeberl
    B D Sun
    T V Damodaran
    T Brown
    D S Millington
    D K Benjamin
    A Bird
    A Schneider
    S Hillman
    M Jackson
    R M Beaty
    Y T Chen
    Gene Therapy, 2006, 13 : 1281 - 1289
  • [26] Development of Helper-Dependent Adenovirus (HDAd) Vectors for Gene Therapy in the Mouse Model for Glycogen Storage Disease Type Ia (GSD-Ia)
    Koeberl, Dwight D.
    Banerjee, Lopamudra
    Sun, Baodong
    Bird, Andrew
    Chen, Y. T.
    Hiro, Kazuhiro
    Chan, Lawrence
    MOLECULAR THERAPY, 2006, 13 : S81 - S82
  • [27] Long-Term Efficacy and Safety in Adults with Glycogen Storage Disease Type IA (GSD IA) from a Phase 1/2 Clinical Trial and Long-Term Follow-Up Study of DTX401, an AAV8-Mediated, Liver-Directed Gene Therapy
    Mitchell, John J.
    Riba-Wolman, Rebecca
    Rodriguez-Buritica, David
    Ahmad, Ayesha
    Pico, Maria-Luz Couce
    Derks, Terry G.
    Weinstein, David A.
    Mitragotri, Deepali
    Collis, Richard
    Grimm, Andrew Alexander
    MOLECULAR THERAPY, 2024, 32 (04) : 78 - 79
  • [28] Erratum: Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia
    D D Koeberl
    B D Sun
    T V Damodaran
    T Brown
    D S Millington
    D K Benjamin
    A Bird
    A Schneider
    S Hillman
    M Jackson
    R M Beaty
    Y T Chen
    Gene Therapy, 2007, 14 : 281 - 281
  • [29] Erratum: Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia
    D D Koeberl
    B D Sun
    T V Damodaran
    T Brown
    D S Millington
    D K Benjamin
    A Bird
    A Schneider
    S Hillman
    M Jackson
    R M Beaty
    Y T Chen
    Gene Therapy, 2006, 13 : 1430 - 1430
  • [30] Gene therapy using a novel G6PC-S298C variant enhances the long-term efficacy for treating glycogen storage disease type Ia
    Zhang, Lisa
    Lee, Cheol
    Arnaoutova, Irina
    Anduaga, Javier
    Starost, Matthew F.
    Mansfield, Brian C.
    Chou, Janice Y.
    BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, 2020, 527 (03) : 824 - 830