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

被引：0

作者：

Harding, Cary O. ^{[1
]}

Lin, Julie ^{[2
]}

Crombez, Eric ^{[2
]}

机构：

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

[2] Ultragenyx Gene Therapy, Cambridge, MA USA

来源：

MOLECULAR GENETICS AND METABOLISM | 2018年 / 123卷 / 03期

关键词：

D O I：

暂无

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

引用

页码：198 / 198

页数：1

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