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：

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

页数：3