AAV8-Mediated Liver-Directed Gene Therapy as a Potential Therapeutic Option in Adults with Glycogen Storage Disease Type Ia (GSDIa): Results from a Phase 1/2 Clinical Trial

被引：0
|
作者：
Weinstein, David A. [1 ]
Ahmad, Ayesha [2 ]
Rodriguez-Buritica, David F. [3 ]
Mitchell, John [4 ]
Derks, Terry G. [5 ]
Mou, Jiani [6 ]
Poma, Allen [6 ]
Crombez, Eric [6 ]
机构：
[1] Univ Connecticut, Farmington, CT USA
[2] Univ Michigan, Ann Arbor, MI 48109 USA
[3] Univ Texas McGovern Med Sch, Houston, TX USA
[4] Montreal Childrens Hosp, Montreal, PQ, Canada
[5] Univ Groningen, Groningen, Netherlands
[6] Ultragenyx Gene Therapy, Cambridge, MA USA
来源：
MOLECULAR THERAPY | 2020年 / 28卷 / 04期
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：
1306
引用
收藏
页码：558 / 558
页数：1
相关论文
共 41 条
  • [11] Sustained Efficacy and Safety at Week 52 and Up to Three Years in Adults with Glycogen Storage Disease Type iA (GSDIa): Results from a Phase 1/2 Clinical Trial of DTX401, an AAV8-mediated, Liver-Directed Gene Therapy
    Riba-Wolman, Rebecca
    Rodriguez-Buritica, David F.
    Ahmad, Ayesha
    Pico, Maria-Luz Couce
    Derks, Terry G.
    Mitchell, John
    Weinstein, David A.
    Mitragotri, Deepali
    Valayannopoulos, Vassili
    Crombez, Eric
    MOLECULAR THERAPY, 2022, 30 (04) : 564 - 564
  • [12] Sustained efficacy and safety up to 3.5 years in adults with glycogen storage disease type Ia (GSDIa): longer-term results from a phase 1/2 clinical trial of DTX401, an AAV8-mediated, liver-directed gene therapy
    Mitchell, J.
    Derks, T. G.
    Riba-Wolman, R.
    Rodriguez-Buritica, D. F.
    Ahmad, A.
    Pico, M. L. Couce
    Weinstein, D. A.
    Mitragotri, D.
    Crombez, E.
    HUMAN GENE THERAPY, 2022, 33 (23-24) : A25 - A26
  • [13] Efficacy and Safety at Week 52 and up to Four Years in Adults with Glycogen Storage Disease Type IA (GSDIa): Results from a Phase 1/2 Clinical Trial and Long-Term Follow-Up Study of DTX401, an AAV8-Mediated, Liver-Directed Gene Therapy
    Riba-Wolman, Rebecca
    Rodriguez-Buritica, David F.
    Ahmad, Ayesha
    Couce Pico, Maria-Luz
    Derks, Terry G.
    Mitchell, John
    Weinstein, David A.
    Mitragotri, Deepali
    Grimm, Andrew Alexander
    Crombez, Eric
    MOLECULAR THERAPY, 2023, 31 (04) : 2 - 3
  • [14] A phase 1/2 clinical trial for AAV8-mediated liver-directed gene therapy in adults with late-onset OTC deficiency
    Geberhiwot, T.
    Aldamiz-Echevarria, L.
    Couce, M. L.
    Harding, C. O.
    Khan, A.
    Tan, W. H.
    Lin, J.
    Crombez, E.
    Diaz, G. A.
    HUMAN GENE THERAPY, 2018, 29 (12) : A59 - A59
  • [15] Liver-Directed AAV Gene Transfer for Glycogen Storage Disease Type IA
    Jauze, Louisa
    Miliano, Alexane Cannella
    Silva, Marine
    Zitoun, Carine
    Mithieux, Gilles
    Ronzitti, Giuseppe
    Rajas, Fabienne
    MOLECULAR THERAPY, 2021, 29 (04) : 238 - 238
  • [16] A PHASE 1/2 CLINICAL TRIAL FOR AAV8-MEDIATED LIVER-DIRECTED GENE THERAPY IN ADULTS WITH LATE-ONSET OTC DEFICIENCY: INTERIM RESULTS FROM COHORT 1
    Harding, Cary O.
    Geberhiwot, Tarekegn
    Aldamiz-Echevarria, Luis
    Luz Couce, Maria
    Khan, Aneal
    Tan, Wen-Hann
    Crombez, Eric
    Diaz, George A.
    MOLECULAR GENETICS AND METABOLISM, 2019, 127 (03) : 240 - 240
  • [17] A PHASE 1/2 CLINICAL TRIAL FOR AAV8-MEDIATED LIVER-DIRECTED GENE THERAPY IN ADULTS WITH LATE-ONSET OTC DEFICIENCY: INTERIM RESULTS FROM COHORT 1
    Harding, Cary O.
    Geberhiwot, Tarekegn
    Aldamiz-Echevarria, Luis
    Luz Couce, Maria
    Khan, Anal
    Tan, Wen-Hann
    Crombez, Eric
    Diaz, George A.
    MOLECULAR GENETICS AND METABOLISM, 2019, 126 (03) : 278 - 278
  • [18] 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
  • [19] Preliminary Results from Cohorts 1 and 2 of CAPtivate: A Phase 1/2 Clinical Trial of AAV8-Mediated Liver-Directed Gene Therapy in Adults with Late-Onset OTC Deficiency
    Diaz, George A.
    Harding, Cary O.
    Geberhiwot, Tarekegn
    Aldamiz-Echevarria, Luis
    Luz Couce, Maria
    Khan, Aneal
    Tan, Wen-Hann
    Crombez, Eric
    MOLECULAR THERAPY, 2019, 27 (04) : 2 - 3
  • [20] DESIGN OF AN OPEN LABEL PHASE 1/2 CLINICAL TRIAL FOR AAV8-MEDIATED LIVER-DIRECTED GENE THERAPY IN ADULTS WITH LATE ONSET ORNITHINE TRANSCARBAMYLASE DEFICIENCY
    Harding, Cary O.
    Lin, Julie
    Crombez, Eric
    MOLECULAR GENETICS AND METABOLISM, 2018, 123 (03) : 198 - 198
12345