Cord blood and bone marrow transplantation in inherited metabolic diseases: scientific basis, current status and future directions.

摘要: Progressive degeneration of the central nervous system leading to loss neuromotor, neurophysiological and cognitive abilities is fundamental clinical problem in patients with many inherited metabolic diseases (IMD). Worldwide experience shows that morbidity, quality life, survival these can be improved by allogeneic haematopoietic stem cell transplantation (HSCT), particularly when performed early course disease. At present, while available for some conditions, exogenous enzyme replacement therapy unable correct disease because its inability cross blood-brain barrier. In contrast, HSCT allows donor-derived, enzyme-producing cells migrate brain other organs providing a permanent therapy. may also mediate non-hematopoietic regeneration or repair. Traditionally, bone marrow has been graft source IMD patients. However, last 5 years studies utilizing unrelated donor umbilical cord blood (UCB) as have demonstrated UCB provides rapid increased access favourable outcomes. This review describes preclinical past present treatment approaches discusses current controversies future directions this promising field.