"Bone Marrow Transplantation in Acid Sphingomyelinase-Deficient Mice: Engraftment and Cell Migration into the Brain as a Function of Radiation, Age, and Phenotype."

According to the authors' abstract of an article published in Blood, "Types A and B Niemann-Pick disease (NPD) result from the deficient activity of the lysosomal hydrolase, acid sphingomyelinase (ASM). A long-term goal of our research is to evaluate the effects of bone marrow transplantation (BMT) and hematopoietic stem cell gene therapy (HSCGT) on the NPD phenotype. As an initial step toward this goal, we have undertaken a study aimed at optimizing hematopoietic cell engraftment in acid sphingomyelinase 'knock-out' (ASMKO) mice. Several parameters were analyzed, including the effects of radiation and donor cell number on survival and engraftment of newborn and adult animals, the...

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