A phase 1/2 clinical trial of AAV8 gene therapy in adults with late-onset OTC deficiency: CAPtivate cohort 1+2 results

被引：0
|
作者：
Geberhiwot, T. [1 ]
Aldamiz-Echevarria, L. [2 ]
Couce, M. L. [3 ]
Diaz, G. A. [4 ]
Harding, C. O. [5 ]
Khan, A. [6 ]
Tan, W. H. [7 ]
Lee, C. [8 ]
Puga, A. C. [8 ]
Crombez, E. [8 ]
机构：
[1] Univ Birmingham, Birmingham, W Midlands, England
[2] Cruces Univ Hosp, Biocruces Bizkaia Hlth Res Inst, Baracaldo, Spain
[3] Univ Santiago de Compostela, Santiago De Compostela, Spain
[4] Icahn Sch Med Mt Sinai, New York, NY 10029 USA
[5] Oregon Hlth & Sci Univ, Portland, OR 97201 USA
[6] Univ Calgary, Calgary, AB, Canada
[7] Harvard Med Sch, Boston Childrens Hosp, Boston, MA 02115 USA
[8] Ultragenyx Gene Therapy, Novato, CA USA
来源：
HUMAN GENE THERAPY | 2019年 / 30卷 / 11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：
OR49
引用
收藏
页码：A18 / A18
页数：1
相关论文
共 50 条
  • [21] AN OPEN LABEL PHASE 1/2 CLINICAL TRIAL FOR AAV8-MEDIATED LIVER-DIRECTED GENE THERAPY IN ADULTS WITH GLYCOGEN STORAGE DISEASE TYPE IA
    Weinstein, David A.
    Ahmad, Ayesha
    Poma, Allen
    Crombez, Eric
    MOLECULAR GENETICS AND METABOLISM, 2019, 127 (03) : 309 - 309
  • [22] AN OPEN LABEL PHASE 1/2 CLINICAL TRIAL FOR AAV8-MEDIATED LIVER-DIRECTED GENE THERAPY IN ADULTS WITH GLYCOGEN STORAGE DISEASE TYPE IA
    Weinstein, David A.
    Ahmad, Ayesha
    Poma, Allen
    Crombez, Eric
    MOLECULAR GENETICS AND METABOLISM, 2019, 126 (03) : 333 - 333
  • [23] Regional Venous Infusion of AAV1 or AAV8 Gene Therapy Vectors Results in Robust Muscle Transgene Expression in Rhesus Macaques
    Gruntman, Alisha M.
    Gernoux, Gwladys
    Wang, Gensheng
    Benson, Janet
    Chulay, Jeff
    Knop, Dave
    Mueller, Christian
    Flotte, Terence R.
    MOLECULAR THERAPY, 2017, 25 (05) : 284 - 284
  • [24] 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
    Weinstein, David A.
    Ahmad, Ayesha
    Rodriguez-Buritica, David F.
    Mitchell, John
    Derks, Terry G.
    Mou, Jiani
    Poma, Allen
    Crombez, Eric
    MOLECULAR THERAPY, 2020, 28 (04) : 558 - 558
  • [25] AAV8-Mediated Liver-Directed Gene Therapy as a Potential Therapeutic Option in Adults with Glycogen Storage Disease Type Ia (GSDIa): Updated Phase 1/2 Clinical Trial Results
    Rodriguez-Buritica, David F.
    Ahmad, Ayesha
    Pico, Mari Luz Couce
    Derks, Terry G.
    Mitchell, John
    Riba-Wolman, Rebecca
    Mou, Jiani
    Poma, Allen
    Valayannopoulos, Vassili
    Crombez, Eric
    MOLECULAR THERAPY, 2021, 29 (04) : 134 - 135
  • [26] Phase 1/2 trial of AXO-AAV-GM1 (AAV9-GLB1) gene therapy for infantile- and juvenile-onset GM1 gangliosidosis
    Tifft, Cynthia
    D'Souza, Precilla
    Johnston, Jean M.
    Acosta, Maria T.
    Nicoli, Elena-Raluca
    Rothermel, Caroline
    Thurm, Audrey
    Karunakara, Ajith
    Thorp, Benjamin
    Ross, Peter
    Jameson, John
    Sheehan, Michael
    Vaughn, Toby
    Valencia, Donna
    De Boever, Erika
    Corcoran, Gavin
    MOLECULAR GENETICS AND METABOLISM, 2022, 135 (02) : S119 - S119
  • [27] SUMMARY OF SCREENING DATA FROM 19 PATIENTS WITH LATE-ONSET POMPE DISEASE FOR A PHASE I CLINICAL TRIAL OF AAV VECTOR-MEDIATED GENE THERAPY
    Hannah, William B.
    Case, Laura E.
    Walters, Crista
    Kishnani, Priya S.
    Koeberl, Dwight D.
    MOLECULAR GENETICS AND METABOLISM, 2019, 127 (03) : 274 - 274
  • [28] AXO-AAV-GM2 Gene Therapy for the Treatment of GM2 Gangliosidosis: Preliminary Results from a Phase 1/2 Trial
    Eichler, F.
    Flotte, T.
    Andonian, H.
    Cataltepe, O.
    Artinian, R.
    Nagy, A.
    Thorp, B.
    Jameson, J.
    Sheehan, M.
    Vaughn, T.
    Valencia, D.
    De Boever, E.
    ANNALS OF NEUROLOGY, 2022, 92 : S169 - S170
  • [29] SUMMARY OF SCREENING DATA FROM 19 PATIENTS WITH LATE-ONSET POMPE DISEASE FOR A PHASE I CLINICAL TRIAL OF AAV VECTOR-MEDIATED GENE THERAPY
    Hannah, William B.
    Case, Laura E.
    Walters, Crista
    Kishnani, Priya S.
    Koeberl, Dwight D.
    MOLECULAR GENETICS AND METABOLISM, 2019, 126 (03) : 305 - 306
  • [30] A phase 1 study of gene therapy with ACTUS-101 in late-onset Pompe disease
    Koeberl, Dwight
    Smith, Edward C.
    Case, Laura E.
    Hopkins, Sam
    Childers, Martin K.
    Walters, Crista
    Han, Sang-oh
    Spears, Tracy G.
    Hornik, Christoph P.
    Bali, Deeksha
    Kishnani, Priya S.
    MOLECULAR GENETICS AND METABOLISM, 2020, 129 (02) : S91 - S91
12345