Framework for the Selection of Subjects in Human Gene Therapy and Diagnostic Trials

Grossman M , Raper SE , Kozarsky K , Stein EA , Engelhardt JF , Muller D , Lupien PJ , Wilson JM. Successful ex vivo gene therapy directed to liver in a patient with familial hypercholesterolaemia. Nat Genet 1994;6:335–41.

Wilson JM , Grossman M , Raper SE , Baker JR , Newton RS , Thoene JG. Clinical protocol: ex vivo gene therapy of familial hypercholesterolemia. Hum Gene Ther 1992;3:179–222.

British Medical Association. Our genetic future: the science and ethics of genetic technology. New York: Oxford University Press, 1992.

European Medical Research Councils. Gene therapy in man. Lancet 1988;1(8597):1271–2.

Fost N. Ethical issues in genetics. Pediatr Clin North Am 1992;39:79–89.

Ledley FD. Clinical considerations in the design of protocols for somatic gene therapy. Hum Gene Ther 1991;2:77–83.

Walters L. The ethics of human gene therapy. Nature 1986;320:225–7.

Walters L. Genetics and reproductive technologies. In: Veatch RM , ed. Medical ethics. Boston: Jones and Bartlett, 1989.

Starzl TE , Hakala TR , Tzakis A , Gordon R , Stieber A , Makowka L , Klimoski J , Bahnson HT. A multifactorial system for equitable selection of cadaver kidney recipients. JAMA 1987;257:3073–5.

Bronsther O , Fung JJ , Izakis A , Thiel DV , Starzl TE. Prioritization and organ distribution for liver transplantation. JAMA 1994;271:140–3.

Flavin DK , Niven RG , Kelsey JE. Alcoholism and orthotopic liver transplantation. JAMA 1988;259:1546–7.

Ramos EL , Kasiske BL , Alexander SR , Danovitch GM , Harmon WE , Kahana L , Kiresuk TJ , Neylan JF. The evaluation of candidates for renal transplantation. Transplantation 1994;57:490–7.

Wilson J , Grossman M. Familial hypercholesterolemia. Minutes of the University of Michigan Genome Ethics Advisory Committee; 1993 Jan 26. Ann Arbor. University of Michigan, 1993.

Weber B , Biesecker B , Calzone K. Familial breast cancer. Minutes of the University of Michigan Genome Ethics Advisory Committee; 1993 March 9. Ann Arbor. University of Michigan, 1993.

Surman OS , Purtilo R. Reevaluation of organ transplantation criteria: allocation of scarce resources to borderline candidates. Psychosomatics 1992;33:202–12.

Dossetor JB. Ethical issues in organ allocation. Transplant Proc 1988;(20 Suppl 1):1053–8.

King M. Localization of the early-onset breast cancer gene. Hosp Pract 1991;15:121–6.

Biesecker BB , Boehnke M , Calzone K , Markel DS , Garber JE , Collins FS , Weber BL. Genetic counseling for families with inherited susceptibility to breast and ovarian cancer. JAMA 1993;269:1970–4.

Babul R , Adam S , Kremer B , Dufrasne S , Wiggins S , Huggins M , Theilmann J , Bloch M , Hayden MR , and the Canadian Collaborative Group on Predictive Testing for Huntington Disease. Attitudes toward direct predictive testing for Huntington disease gene. JAMA 1993;270:2321–5.

Beaudet AL , Kazazian HH. Statement from the National Institutes of Health workshop on population screening for the cystic fibrosis gene. N Eng J Med 1990;323:70–1.

Elias S , Kaback MM. Statement of the American Society of Human Genetics on cystic fibrosis carrier screening. Am J Hum Genet 1992;51:1443–4.

Zitelli BJ , Gartner JC , Malatack JJ , Urbach AH , Miller JW , Williams L , Kirkpatrick B , Breinig MK , Ho M. Pediatric liver transplantation: patient evaluation and selection, infectious complications, and life-style after transplantation. Transplant Proc 1987;19:3309–16.

Joint Senate and House Special Committees on Organ Transplantation. Organ transplantation in Michigan. Lansing: Michigan Legislative Service Bureau-Legislative Science Office, 1987:70.

Kilner JF. Selecting patients when resources are limited: a study of U.S. medical directors of kidney dialysis and transplantation facilities. Am J Public Health 1988;78:144–7.

Anderson WF. Genetics and human malleability. Hastings Cent Rep 1990;20:21–4.

Meyers AS. Gene therapy and genetic diseases: revisiting the promise. Hum Gene Ther 1994;5:1201–2.

Ethics Committee of the United Network for Organ Sharing. General principles for allocating human organs. Transplant Proc 1992:24:2227–35.

Samuel D , Benhamou JP , Bismuth H , Gugenheim J , Ciardullo M , Saliba F. Criteria of selection for liver transplantation. Transplant Proc 1987;19:2383–6.

Shaw BW. Exclusion criteria for liver transplant recipients. Transplant Proc 1989;21:3484–6.

Grossman M. The ethical implications of the new genetics. Simulated hypercholesterolemia somatic gene therapy protocol review process; 1994 May 6; Boston. Public Responsibility in Medicine and Research (PRIM&R) and Tufts University School of Medicine, 1994.

Berg K. Predictive genetic testing to control coronary heart disease and hyperlipidemia. Arteriosclerosis 1989;9(1 Suppl):50–8.

Bernhardt BA. A survey of reimbursement for cystic fibrosis carrier testing. J Genet Counseling 1993;2:69–76.

Wilfond BS , Fost N. The cystic fibrosis gene: medical and social implications for heterozygote detection. JAMA 1990;263:2777–83.

Garber AM , Fenerty JP. Costs and benefits of prenatal screening for cystic fibrosis. Med Care 1991;29:473–89.

Daniel J , Callender CO , Flores J , Toussaint R , Yeager C , Bond O , Gear JC. Renal transplantation in substance abusers revisited: the Howard University Hospital experience. Transplant Proc 1989;21:1422–4.

Lucey MR , Merion RM , Henley KS , Campbell DA , Turcotte JG , Nostrant TT , Blow FC , Beresford TP. Selection for and outcome of liver transplantation in alcoholic liver disease. Gastroenterology 1992;102:1736–41.

Lucey MR , Beresford TP. Alcoholic liver disease: to transplant or not to transplant? Alcohol Alcohol 1992;27:103–8.

Cohen C , Benjamin M , and the Ethics and Social Impact Committee of the Transplant and Health Policy Center, Ann Arbor, Michigan. Alcoholics and liver transplantation. JAMA 1991;265:1299–301.

National Research Service Award Act of 1974; Public Law 93-348, 93rd Cong., 2nd Sess. 342 (1974).

US Department of Health Education and Welfare. The Belmont Report: ethical principles for the protection of human subjects of research. National Commission for the Protection of Human Subjects of Biomedical and Behavioral Research. DHEW 1978 Sept. Publ. no. [OS] 78-0012.

National Institutes of Health. Points to consider in the design and submission of human somatic-cell gene therapy protocols. Bethesda (MD): NIH; 1985;8:116–22.

Knoppers B , Laberge C. DNA sampling and informed consent. Can Med Assoc J 1989;140:1023–8.

Caplan AL. Problems in the policies and criteria used to allocate organs for transplantation in the United States. Transplant Proc 1989;21:3381–7.

Craufurd D , Tyler A. Predictive testing for Huntington's disease: protocol of the UK Huntington's Prediction Consortium. J Med Genet 1992;29:915–8.

MacKay CR , Shea JM. Ethical considerations in research on Huntington's disease. Clin Res 1977;25:241–7.

Office of Technology Assessment. New developments in biotechnology, vol. 1. Washington, DC: US GPO 1987:93–103.

A small step for gene therapy [editorial]. Nat Genet 1994;6:323–4.

Caskey CT , Kaback MM , Beaudet AL. The American Society of Human Genetics statement on cystic fibrosis screening. Am J Hum Genet 1990;46:393.

Thies U , Bockel B , Bochdalofsky V. Attitudes of neurologists, psychiatrists, and psychotherapists towards predictive testing for Huntington's disease in Germany. J Med Genet 1993;30:1023–7.

DeGrazia D. The ethical justification for minimal paternalism in the use of the predictive test for Huntington's disease. J Clin Ethics 1991;2:219–28.

Freedman B. Scientific value and validity as ethical requirements for research: a proposed explication. IRB 1987;9:7–10.

Morreim EH. Patient-funded research: paying the piper or protecting the patient? IRB 1991;13:1–6.

Bick D , Black SH , Cummings E , Costakos D , Maddalena A , Headrick EG , Jones SL , Becker R , Schulman JD. Prenatal screening for delta F508 mutation in population not selected for cystic fibrosis. Lancet 1990;336:1324–5.

National Institutes of Health. Ethical, legal, and social implications program activities report. Bethesda (MD): National Center for Human Genome Research; 1993:32.

Department of Energy. Human genome 1991-92 program report. Washington (DC): Office of Health and Environmental Research; 1992:248.

US Department of Health and Human Services. NIH guide for grants and contracts. Washington (DC): 1993 Jan. 22:3, Part II.

Jenks S. RAC approves policy for single-patient use of gene therapy. J Natl Cancer Inst 1993; 85:266–7.

Marshall E. The politics of breast cancer. Science 1993;259:616–7.

Halasz NA. Medicine and ethics: how to allocate transplantable organs. Transplantation 1991;52:43–6.