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Gene therapy for children with cystic fibrosis—who has the right to choose?
  1. A Jaffé1,
  2. S A Prasad1,
  3. V Larcher2,
  4. S Hart3
  1. 1Portex Respiratory Medicine Group, Great Ormond Street Hospital for Children, NHS Trust and Institute of Child Health, London, UK
  2. 2Consultant in General Paediatrics and Ethics, Great Ormond Street Hospital for Children, NHS Trust, London, UK
  3. 3Wolfson Centre for Gene Therapy of Childhood Disease, Institute of Child Health, London, UK
  1. Correspondence to:
 Dr Adam Jaffé MD FRCPCH, FRCP
 Consultant and Honorary Senior Lecturer in Respiratory Research, Portex Respiratory Medicine Group, Level 6 Cardiac Wing, Great Ormond Street Hospital for Children NHS Trust and Institute of Child Health, Great Ormond Street, London, WC1N 3JH, UK; a.jaffe{at}

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It may be unethical to deny children with cystic fibrosis access to ethically approved clinical trials from which they might benefit

Despite advances in nutritional management, aggressive antibiotic usage, and physiotherapy, cystic fibrosis (CF) remains a life limiting illness with high morbidity that imposes considerable burdens on children and families.1 Although survival to 40 years is predicted for children born in 1990s, the median age of death in 2003 was 24.2 years (UK CF Trust database).

The pathophysiological features of CF are produced by a defective gene on chromosome 7, resulting in the defective production of a protein that regulates cellular ion transport. Defective ion transport is thought to lead to increased mucus viscosity (sticky sputum), with poor airway clearance, recurrent bacterial infection, lung damage, and death.

Gene therapy (GT), the insertion of a normally functioning gene into deficient host cells using a suitable vector, is a potential treatment or cure for diseases produced by single gene defects—for example, CF. Gene therapy does, however, have potential or actual risks, leading many to suggest that evidence of efficacy in adults should be demonstrated before trials are conducted in children. Many serious diseases in adults such as CF have their onset in childhood. If early treatment provides greater hopes of benefit, children may be more appropriate targets for GT in CF than adults. It may be unethical to deny them access to properly constructed, ethically approved clinical trials from which they might benefit.

Since research in children should be scientifically valid, in the child’s best interests, and the subject of valid consent, this article will consider these parameters in relation to trials of GT in children with CF. Because of the importance of consumer participation in the design of research we present the results of a questionnaire about GT trials delivered …

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  • Competing interests: Dr Hart has a stake in Genex Biosystems Ltd, a company that develops vectors.