Marrow Transplantation in Genetic Disease

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Abstract
Recent experience with bone-marrow transplantation for the treatment of inherited disorders of metabolism has highlighted several complicated issues regarding the procedure.1 2 3 4 The report by Krivit et al.4 in this issue of the Journal adds another case to the series and provides a few more pieces of important data related to the general question of therapy for genetic disorders of metabolism. Because many aspects of the pathogenesis of these diseases are undefined, physicians are constantly in dialogue with basic scientists studying the biochemistry, cell biology, and molecular genetics of these disorders. The conclusions of these discussions must result in the answer . . .