TY - JOUR AU - Proia, Richard L. AU - Wu, Yun-Ping T1 - Blood to brain to the rescue PY - 2004/04/15/ AB - Neurodegeneration occurs in the majority of the more than 40 known lysosomal storage diseases. Since the nervous system in these disorders can be globally affected, effective treatment would require persistent widespread correction. Biffi et al. show such correction is possible in a mouse model of metachromatic leukodystrophy by the transplantation of hematopoietic cells genetically modified to overexpress the missing lysosomal enzyme. The results reveal a nervous system damage-response pathway that can be harnessed to provide therapy to the nervous system in these serious disorders. JF - The Journal of Clinical Investigation JA - J Clin Invest SN - 0021-9738 DO - 10.1172/JCI21476 VL - 113 IS - 8 UR - https://doi.org/10.1172/JCI21476 SP - 1108 EP - 1110 PB - The American Society for Clinical Investigation ER -