针对囊性纤维化患儿的基因治疗——谁有权做出选择？
Gene therapy for children with cystic fibrosis--who has the right to choose?
作者信息
Jaffé A, Prasad S A, Larcher V, Hart S
机构信息
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.
出版信息
J Med Ethics. 2006 Jun;32(6):361-4. doi: 10.1136/jme.2005.012740.
Abstract
It may be unethical to deny children with cystic fibrosis access to ethically approved clinical trials from which they might benefit
摘要
拒绝囊性纤维化患儿参与经伦理批准且他们可能从中受益的临床试验，这可能是不道德的。
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本文引用的文献
1
Guidelines for the ethical conduct of medical research involving children.
Bull Med Ethics. 1992 Jul-Aug;80:13-20.
2
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 Dec;15(12):1255-69. doi: 10.1089/hum.2004.15.1255.
3
Recent developments in gene transfer: risk and ethics.
BMJ. 2005 Jan 8;330(7482):79-82. doi: 10.1136/bmj.330.7482.79.
4
Increasing response rates to postal questionnaires: a randomised trial of variations in design.
J Health Serv Res Policy. 2004 Oct;9(4):213-7. doi: 10.1258/1355819042250159.
5
Gene therapy for cystic fibrosis: an example for lung gene therapy.
Gene Ther. 2004 Oct;11 Suppl 1(Suppl 1):S43-50. doi: 10.1038/sj.gt.3302368.
6
Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: a multicenter, double-blind, placebo-controlled trial.
Chest. 2004 Feb;125(2):509-21. doi: 10.1378/chest.125.2.509.
7
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1.
Science. 2003 Oct 17;302(5644):415-9. doi: 10.1126/science.1088547.
8
Gene therapy: ethical issues.
Theor Med Bioeth. 2003;24(1):31-58. doi: 10.1023/a:1022967623162.
9
Informed consent for pediatric research: is it really possible?
J Pediatr. 2003 Feb;142(2):89-90. doi: 10.1067/mpd.2003.64.
10
Evaluation of a porcine model for pulmonary gene transfer using a novel synthetic vector.
J Gene Med. 2002 Jul-Aug;4(4):438-46. doi: 10.1002/jgm.270.
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