Early Clinical Trial Results Following Administration of a Low Dose of a Novel Self Complementary Adeno-Associated Viral Vector Encoding Human Factor IX In Two Subjects with Severe Hemophilia B

被引：16

作者：

Nathwani, Amit ^{[1
]}

Tuddenham, Edward ^{[3
]}

Rosales, Cecilia ^{[1
]}

McIntosh, Jenny ^{[1
]}

Riddell, Anne ^{[2
,3
]}

Rustagi, Pradip ^{[4
]}

Glader, Bertil ^{[4
]}

Kay, Mark ^{[4
]}

Allay, James ^{[5
]}

Coleman, John ^{[5
]}

Sleep, Susan ^{[5
]}

High, Katherine A. ^{[6
]}

Mingozzi, Federico ^{[6
]}

Gray, John T.

Reiss, Ulrike M.

Nienhuis, Arthur W.

Davidoff, Andrew ^{[7
]}

机构：

[1] UCL, London, England

[2] Royal Free NHS Trust, KD Haemophilia Ctr, London, England

[3] Royal Free NHS Trust, Thrombosis Unit, London, England

[4] Stanford Univ, Sch Med, Stanford, CA 94305 USA

[5] St Jude Childrens Hosp, Childrens GMP, LLC, Memphis, TN 38105 USA

[6] Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA

[7] St Jude Childrens Hosp, Dept Surg, Memphis, TN 38105 USA

来源：

关键词：

D O I：

10.1182/blood.V116.21.248.248

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

引用

页码：114 / 114

页数：1