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

Clinical Medicine

Volumn 4, Issue 1, 2004, Pages 54-56

  Ludlam, C A ^a  

^a ROYAL INFIRMARY OF EDINBURGH   (United Kingdom)

Author keywords

Adverse events; Ethics; Gene therapy; Haemophilia; Legislation

Indexed keywords

ADENOVIRUS VECTOR; ORNITHINE CARBAMOYLTRANSFERASE;

DISORDERS OF AMINO ACID AND PROTEIN METABOLISM; GENE THERAPY; HEMOPHILIA; HUMAN; MEDICAL ETHICS; MEDICAL RESEARCH; MULTIPLE ORGAN FAILURE; REGISTER; REVIEW; RISK ASSESSMENT; UNITED KINGDOM;

EID: 1342290697     PISSN: 14702118     EISSN: None     Source Type: Journal    
DOI: 10.7861/clinmedicine.4-1-54     Document Type: Review

References (10)

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3. Marshall E. Gene therapy death prompts review of adenovirus vector. Science 1999;286:2244-5.
4. Hacein-Bey-Abina S, von Kalle C, Schmidt M, Le Deist F et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2003;348:255-6.
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9. Powell JS, Ragni MV, White GC, Lusher J et al. Phase I trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous injection. Blood 2003;102:2038-45.
10. www.doh.gov.uk/genetics/gtac.