GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant

被引：31
|
作者：
Nathwani, Amit C. [1 ,2 ,3 ,4 ]
Tuddenham, Edward [5 ]
Chowdary, Pratima [6 ]
McIntosh, Jenny [4 ]
Lee, Doyoung [5 ]
Rosales, Cecilia [5 ]
Phillips, Mark [7 ]
Pie, Jun [8 ]
Zhou Junfang [9 ]
Meagher, Michael M. [10 ]
Reiss, Ulrike [11 ]
Davidoff, Andrew M. [12 ]
Morton, Chris L. [9 ]
Riddell, Anne [2 ,3 ]
机构：
[1] Freeline, Stevenage, Herts, England
[2] Royal Free NHS Fdn Trust, Katharine Dormandy Haemophilia Ctr, London, England
[3] Royal Free NHS Fdn Trust, Thrombosis Unit, London, England
[4] UCL, Canc Inst, Dept Haematol, London, England
[5] UCL Canc Inst, Dept Haematol, London, England
[6] Royal Free Hosp, KD Haemophilia & Thrombosis Ctr, London, England
[7] UCL, UCL Canc Inst, London, England
[8] KD Haemophilia & Thrombosis Ctr, London, England
[9] St Jude Childrens Res Hosp, Dept Surg, 332 N Lauderdale St, Memphis, TN 38105 USA
[10] St Jude Childrens Res Hosp, Childrens GMP LLC, 332 N Lauderdale St, Memphis, TN 38105 USA
[11] St Jude Childrens Rsch Hosp, Dept Surg, Memphis, TN USA
[12] St Jude Childrens Res Hosp, 332 N Lauderdale St, Memphis, TN 38105 USA
来源：
BLOOD | 2018年 / 132卷
关键词：
D O I：
10.1182/blood-2018-99-118256
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
489
引用
收藏
页数：3
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