Our Journey to Successful Gene Therapy for Hemophilia B

被引：12

作者：

Nathwani, Amit C. ^{[1
,2
,3
,4
]}

Nienhuis, Arthur W. ^{[5
]}

Davidoff, Andrew M. ^{[6
]}

机构：

[1] Royal Free NHS Fdn Trust, Katharine Dormandy Haemophilia Ctr, London NW3 2QG, England

[2] Royal Free NHS Fdn Trust, Thrombosis Unit, London NW3 2QG, England

[3] UCL Canc Inst, Dept Haematol, London WC1E 6DD, England

[4] Natl Hlth Serv Blood & Transplant, Borehamwood WD24 4QN, Herts, England

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

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

来源：

HUMAN GENE THERAPY | 2014年 / 25卷 / 11期

基金：

英国医学研究理事会;

关键词：

ADENOASSOCIATED VIRUS VECTORS; COAGULATION-FACTOR-IX; HIGHLY EFFICIENT TRANSDUCTION; NONHUMAN PRIMATE MODELS; HIGH-LEVEL EXPRESSION; IN-VIVO; VIRAL VECTOR; AAV VECTOR; CAPSID PROTEINS; HUMAN FIX;

D O I：

10.1089/hum.2014.2540

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

[No abstract available]

引用

页码：923 / 926

页数：4

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