Stable Factor IX Activity Following AAV-Mediated Gene Transfer in Patients with Severe Hemophilia B

被引：1

作者：

Davidoff, Andrew ^{[1
]}

Tuddenham, Edward G. D. ^{[2
,3
]}

Rangarajan, Savita ^{[4
]}

Rosales, Cecilia

McIntosh, Jenny

Chowdary, Pratima ^{[3
,6
]}

Riddell, Anne ^{[2
,3
]}

Glader, Bertil ^{[7
]}

Rustagi, Pradip ^{[8
]}

Ng, Catherine ^{[1
]}

Kay, Mark ^{[8
]}

Zhou, Junfang ^{[1
]}

Spence, Yunyu ^{[9
]}

Morton, Christopher ^{[10
]}

Allay, James ^{[11
]}

Sleep, Susan ^{[11
]}

Basner-Tschakarjan, Etiena

Mingozzi, Federico ^{[12
]}

High, Katherine A.

Gray, John T.

Reiss, Ulrike M. ^{[9
]}

Nienhuis, Arthur W. ^{[9
]}

Nathwani, Amit C. ^{[5
]}

机构：

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

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

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

[4] Guys & St Thomas NHS Fdn Trust, Ctr Haemostasis & Thrombosis, London, England

[5] UCL Canc Inst, Dept Haematol, London, England

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

[7] Stanford Univ, Sch Med, Palo Alto, CA 94304 USA

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

[9] St Jude Childrens Res Hosp, Dept Hematol, Memphis, TN 38105 USA

[10] St Jude Childrens Res Hosp, Memphis, TN USA

[11] Childrens GMP LLC, St Jude Childrens Res Hosp, Memphis, TN USA

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

来源：

BLOOD | 2012年 / 120卷 / 21期

关键词：

D O I：

10.1182/blood.V120.21.752.752

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

752

引用

页数：2

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