Bone marrow transplantation in newborn rats with mucopolysaccharidosis type VI: biochemical, pathological, and clinical findings

Transplantation
C M SimonaroE H Schuchman

Abstract

Mucopolysaccharidosis type VI (MPS VI) is the lysosomal storage disorder caused by the deficient activity of arylsulfatase B (ASB). In this study, we evaluated bone marrow transplantation (BMT) for the treatment of MPS VI and the effects of irradiation on the survival and engraftment of bone marrow-transplanted neonatal rats. One- to 2-day-old MPS VI rats were injected with normal bone marrow after irradiation with 200, 400, or 800 cGy. Ninety percent of the animals receiving a single dose of 200 cGy (n=30) survived the procedure, whereas irradiation with 400 cGy (n=23) or 800 cGy (n=12) resulted in significant mortality (78% and 100%, respectively). Engraftment was monitored by determining ASB activities in peripheral white blood cells and by Y chromosome in situ hybridization analysis. Fifty-two percent of the animals from the 200-cGy group engrafted for up to 8 months after BMT; among the five animals that survived the 400-cGy dose, all engrafted. In comparison, only 20% of nonirradiated animals engrafted at low levels. Of the 24 engrafted animals that were monitored for 8 months after BMT, clinical and/or radiographic improvements were noted in only one (BMT animal 3). Enzymatic analysis revealed that the ASB activities in ...Continue Reading

References

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Citations

Mar 27, 2001·Transplantation Proceedings·P A AndrewsR W Chang
Mar 8, 2012·Molecular Therapy : the Journal of the American Society of Gene Therapy·Katherine P PonderMark E Haskins
Oct 24, 2007·Molecular Therapy : the Journal of the American Society of Gene Therapy·Alessandra TessitoreAlberto Auricchio
Aug 9, 2003·Pediatric Research·Oliver F StrauchChristoph Peters
Mar 30, 2007·Acta Paediatrica. Supplement·Mark E Haskins
Sep 29, 2011·Molecular Genetics and Metabolism·Guilherme BaldoKatherine P Ponder
Feb 8, 2003·Acta Paediatrica. Supplement·M HaskinsU Giger
Nov 19, 2015·Journal of Inherited Metabolic Disease·Shunji TomatsuMarta Serafini
Mar 10, 1999·Journal of Inherited Metabolic Disease·E HerskhovitzA Vellodi

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