Gene therapy for children with cystic fibrosis - Who has the right to choose?

Journal of Medical Ethics

Volumn 32, Issue 6, 2006, Pages 361-364

  Jaffé, Adam ^a   Prasad, S A ^a   Larcher, V ^b   Hart, S ^c  

^a Great Ormond Street Hospital for Children NHS Trust   (United Kingdom)

^b GREAT ORMOND STREET HOSPITAL   (United Kingdom)

^c UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; IMMUNOSUPPRESSIVE AGENT; LIPOSOME; RETROVIRUS VECTOR; VIRUS VECTOR;

ADVISORY COMMITTEE; ARTICLE; AWARENESS; CHILDHOOD DISEASE; CLINICAL TRIAL; COMBINED IMMUNODEFICIENCY; COMPETENCE; COUGHING; CYSTIC FIBROSIS; DRUG FATALITY; FLU LIKE SYNDROME; GENE THERAPY; HUMAN; ION TRANSPORT; LEUKEMIA; LIFE EXPECTANCY; LUNG DEVELOPMENT; MEDICAL DECISION MAKING; MEDICAL ETHICS; NONHUMAN; ORGAN TRANSPLANTATION; PARENTAL ATTITUDE; PARENTAL CONSENT; PATIENT AUTONOMY; PREPUBERTY; RESEARCH ETHICS; RISK ASSESSMENT; RISK BENEFIT ANALYSIS; SAFETY; TREATMENT OUTCOME; VALIDATION PROCESS; VIRAL GENE DELIVERY SYSTEM; WHEEZING;

ATTITUDE TO HEALTH; CHILD; CLINICAL TRIALS; CYSTIC FIBROSIS; FAMILY; GENE THERAPY; HUMANS; INFORMED CONSENT; PARENTS; PERSONAL AUTONOMY;

EID: 33745218740     PISSN: 03066800     EISSN: None     Source Type: Journal    
DOI: 10.1136/jme.2005.012740     Document Type: Article

References (29)

1. Jaffe A, Bush A. Cystic fibrosis: review of the decade. Monaldi Arch Chest Dis 2001;56:240-7.
2. Moss RB, Rodman D, Spencer T, et al. Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis. Chest 2004;125:509-21.
3. Alton EWFW, Stern M, Farley R, et al. Cationic lipid-medialed CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial. Lancet 1999;353:947-54.
4. Cunningham S, Meng QH, Klein N, et al. Evaluation of a porcine model for pulmonary gene transfer using a novel synthetic vector. J Gene Med 2002;4:438-46.
5. Konstan MW, Davis PB, Wagener JS, et al. Compacted DNA nanoparticles administered to the nasal mucosa of cystic fibrosis subjects are safe and demonstrate partial to complete cystic fibrosis transmembrane regulator reconstitution. Hum Gene Ther 2004;15:1255-69.
6. Griesenbach U, Geddes DM, Alton EW. Gene therapy for cystic fibrosis: an example for lung gene therapy. Gene Ther 2004;11:S43-S50.
7. Khan TZ, Wagener JS, Bost T, et al. Early pulmonary inflammation in infants with cystic fibrosis. Am J Respir Crit Care Med 1995;151:1075-82.
8. Ranganathan SC, Dezateux C, Bush A, et al. Airway function in infants newly diagnosed with cystic fibrosis. Lancet 2001;358:1964-5.
9. Kitson C, Angel B, Judd D, et al. The extra- and intracellular barriers to lipid and adenovirus-mediated pulmonary gene transfer in native sheep airway epithelium. Gene Ther 1999;6:534-46.
10. Rubinstein RC, McVeigh U, Flotte TR, et al. CFTR gene transduction in neonatal rabbits using anadeno-associated virus (AAV) vector. Gene Ther 1997;4:384-92.
11. Jaffe A, Judd D, Ratcliffe C, et al. Cationic lipid-mediated gene transfer to the growing murine and human airway. Gene Ther 2000;7:273-8.
12. Davis PB, Byard PJ, Konstan MW. Identifying treatments that halt progression of pulmonary disease in cystic fibrosis. Pediatr Res 1997;41:161-5.
13. Alderson P, Montgomery J. Healthcare choices. Making decisions with children. London: Institute of Public Policy Research, 1996.
14. Ethics Advisory Committee. Guidelines for the ethical conduct of medical research involving children. London: British Paediatric Association, 1992.
15. McIntosh N, Bates P, Brykczynska G, et al. Guidelines for the ethical conduct of medical research involving children. Royal College of Paediatrics, Child Health: Ethics Advisory Committee, Arch Dis Child 2000;82:177-82.
16. Gene Therapy Advisory Committee. Operational procedures for the gene therapy advisory committee in its role as the national ethics committee for gene therapy clinical trials. London: Department of Health, June 2004.
17. Nuffield Council on Bioethics. Animal to human transplants. Ethics of xenotransplantation. London: Nuffield Council on Bioethics, 1996.
18. Kimmelman J. Recent developments in gene transfer: risk and ethics. BMJ 2005;330:79-82.
19. Hacein-Bey-Abina S, Von Kalle C, Schmidt M, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003;302:415-19.
20. Grundy R, Larcher V, Gosden RG, et al. Fertility preservation for children treated for cancer(2): ethics of consent for gamete storage and experimentation. Arch Dis Child 2001;84:360-2.
21. Snowdon C, Garcia J, Elbourne D. Making sense of randomization; responses of parents of critically ill babies to random allocation of treatment in a clinical trial. Soc Sci Med 1997;45:1337-55.
22. General Assembly of the United Nations Convention on the Rights of the Child, 1989. United Nations Convention of the Rights of the Child. London: The Stationery Office, 1996.
23. Ondrusek N, Abramovitch R, Pencharz P, et al. Empirical examination of the ability of children to consent to clinical research. J Med Ethics 1998;24:158-65.
24. Gillick v W Norfolk, Wisbech AHA. 3 All ER 402 1985.
25. Blubond-Langner M, De Cico A, Belasco J. Involving children with life shortening illness in discussion about participation in clinical research: a proposal for shuttle diplomacy and negotiation. In: Kodish E, ed. Ethics and research with children: a case-based approach. New York: Oxford University Press, 2005:323-43.
26. Rabino I. Gene therapy: ethical issues. Theor Med Bioeth 2003;24:31-58.
27. Puffer S, Porthouse J, Birks Y, et al. Increasing response rates to postal questionnaires: a randomised trial of variations in design. J Health Serv Res Policy 2004;9:213-17.
28. Jaffé A, Bush A, Geddes DM, et al. Prospects for gene therapy in cystic fibrosis. Arch Dis Child 1999;80:286-9.
29. Kodish E. Informed consent for pediatric research: is it really possible? J Pediatr 2003;142:89-90.