Spk-8011: Preliminary Results from a Phase 1/2 Dose Escalation Trial of an Investigational AAV-Mediated Gene Therapy for Hemophilia a

被引：0

作者：

George, Lindsey A.

Ragni, Margaret V.

Samelson-Jones, Ben J.

Cuker, Adam

Runoski, Alexa R.

Cole, Grace

Wright, Fraser

Chen, Yifeng

Hui, Daniel J.

Wachtel, Katie

Takefman, Daniel

Couto, Linda B.

Reape, Kathleen Z.

Carr, Marcus E.

Anguela, Xavier M.

High, Katherine A.

机构：

来源：

BLOOD | 2017年 / 130卷

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：

604

引用

页数：2

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[50] COHORT 1-4 RESULTS FROM A PHASE 1/2 CLINICAL TRIAL OF AN AAV8-MEDIATED LIVER-DIRECTED GENE THERAPY IN ADULTS WITH GLYCOGEN STORAGE DISEASE TYPE IA (GSDIA) [J].

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