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
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