AAV8 Gene Therapy as a Potential Treatment in Adults with Late-Onset Ornithine Transcarbamylase (OTC) Deficiency: Updated Results from a Phase 1/2 Clinical Trial

被引：0

作者：

Harding, Cary O. ^{[1
]}

Luz Couce, Maria ^{[2
]}

Geberhiwot, Tarekegn ^{[3
]}

Tan, Wen-Hann ^{[4
]}

Khan, Aneal ^{[5
]}

Aldamiz-Echevarria, Luis ^{[6
]}

Diaz, George A. ^{[7
]}

Lee, Connie ^{[8
]}

Puga, Ana Cristina ^{[8
]}

Crombez, Eric ^{[8
]}

机构：

[1] Oregon Hlth & Sci Univ, Portland, OR 97201 USA

[2] Univ Santiago de Compostela, Santiago De Compostela, Spain

[3] Univ Birmingham, Birmingham, W Midlands, England

[4] Harvard Med Sch, Boston Childrens Hosp, Boston, MA 02115 USA

[5] Univ Calgary, Calgary, AB, Canada

[6] Cruces Univ Hosp, Biocruces Bizkaia Hlth Res Inst, Baracaldo, Spain

[7] Icahn Sch Med Mt Sinai, New York, NY 10029 USA

[8] Ultragenyx Gene Therapy, Cambridge, MA USA

来源：

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：

引用

页码：17 / 17

页数：1