Gene therapy trials in the UK: is haemophilia a suitable 'model'?

被引：0

作者：

Ludlam, CA ^{[1
]}

Pasi, KJ ^{[1
]}

Collins, PW ^{[1
]}

Maggs, PHBB ^{[1
]}

Cumming, AM ^{[1
]}

Dolan, G ^{[1
]}

Fryer, A ^{[1
]}

Hill, FGH ^{[1
]}

Peake, IR ^{[1
]}

Perry, D ^{[1
]}

Smith, M ^{[1
]}

机构：

[1] Royal Infirm, Dept Haematol, Edinburgh EH3 9YW, Midlothian, Scotland

来源：

CLINICAL MEDICINE | 2004年 / 4卷 / 01期

关键词：

adverse events; ethics; gene therapy; haemophilia; legislation;

D O I：

暂无

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Gene therapy may be the next major advance for treatment of many diseases, and severe haemophilia (an inherited deficiency of coagulation factor VIII or IX) is a useful model. Progress in gene therapy has been slowed down following fatal multi-organ failure during an adenovirus vector trial for ornithine-transcarbamylase deficiency and two episodes of leukaemia in a retroviral vector trial for severe combined immunodeficiency trial. A small number of early haemophilia clinical trials are in progress or reported. This paper considers ethical and statutory issues related to gene therapy for severe haemophilia within the UK and how these can be addressed through a well-established national network of haemophilia centres. It is likely that these issues will be relevant to clinicians considering gene therapy for other diseases.

引用

页码：54 / 56

页数：3

共 9 条

[1] A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency [J].

Hacein-Bey-Abina, S ;

von Kalle, C ;

Schmidt, M ;

Le Deist, F ;

Wulffraat, N ;

McIntyre, E ;

Radford, I ;

Villeval, JL ;

Fraser, CC ;

Cavazzana-Calvo, M ;

Fischer, A .

NEW ENGLAND JOURNAL OF MEDICINE, 2003, 348 (03) :255-256

[2]

Kay MA, 2002, BLOOD, V100, p115A

[3] Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector [J].

Kay, MA ;

Manno, CS ;

Ragni, MV ;

Larson, PJ ;

Couto, LB ;

McClelland, A ;

Glader, B ;

Chew, AJ ;

Tai, SJ ;

Herzog, RW ;

Arruda, V ;

Johnson, F ;

Scallan, C ;

Skarsgard, E ;

Flake, AW ;

High, KA .

NATURE GENETICS, 2000, 24 (03) :257-261

[4] Clinical trials - Gene therapy death prompts review of adenovirus vector [J].

Marshall, E .

SCIENCE, 1999, 286 (5448) :2244-2245

[5] Gene therapy for haemophilia [J].

Pasi, KJ .

BRITISH JOURNAL OF HAEMATOLOGY, 2001, 115 (04) :744-757

[6] Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion [J].

Powell, JS ;

Ragni, MV ;

White, GC ;

Lusher, JM ;

Hillman-Wiseman, C ;

Moon, TE ;

Cole, V ;

Ramanathan-Girish, S ;

Roehl, H ;

Sajjadi, N ;

Jolly, DJ ;

Hurst, D .

BLOOD, 2003, 102 (06) :2038-2045

[7] Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A [J].

Roth, DA ;

Tawa, NE ;

O'Brien, JM ;

Treco, DA ;

Selden, RF .

NEW ENGLAND JOURNAL OF MEDICINE, 2001, 344 (23) :1735-1742

[8] Gene therapy for the hemophilias [J].

VandenDriessche, T ;

Collen, D ;

Chuah, MKL .

JOURNAL OF THROMBOSIS AND HAEMOSTASIS, 2003, 1 (07) :1550-1558

[9]

White GC, 2001, THROMB HAEMOSTASIS, V86, P172

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