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

被引：0

作者：

Diaz, George A. ^{[1
]}

Harding, Cary O. ^{[2
]}

Geberhiwot, Tarekegn ^{[3
]}

Aldamiz-Echevarria, Luis ^{[4
]}

Luz Couce, Maria ^{[5
]}

Khan, Aneal ^{[6
]}

Tan, Wen-Hann ^{[7
]}

Crombez, Eric ^{[8
]}

机构：

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

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

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

[4] Hosp Univ Cruces, Baracaldo, Spain

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

[6] Univ Calgary, Calgary, AB, Canada

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

[8] Ultragenyx Gene Therapy, Cambridge, MA USA

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D O I：

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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：2 / 3

页数：2