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