Confronting the Issues of Therapeutic Misconception, Enrollment Decisions, and Personal Motives in Genetic Medicine-Based Clinical Research Studies for Fatal Disorders

作者: Lisa M. Arkin , Dolan Sondhi , Stefan Worgall , Lily Hyon K. Suh , Neil R. Hackett

DOI: 10.1089/HUM.2005.16.1028

关键词:

摘要: Genetic medicine-based therapies have unlocked the potential for ameliorating diseases previously considered inevitably fatal. Inherent in clinical trials of genetic medicines are ethical issues therapeutic misconception, enrollment decisions as they relate to risks and benefits research, complex relationships among funding sources, investigators, families affected individuals. The purpose this paper is help define these relevant use describe strategy we used confront a phase I trial adeno-associated virus-mediated gene transfer central nervous system children with late infantile neuronal ceroid lipofuscinosis (LINCL), fatal lysosomal storage disease associated progressive neurodegeneration death by mid-childhood. Our approach challenges should provide useful paradigm investigators initiating other medicine- based studies treat fa...

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