Effects of bone marrow transplantation on the cardiovascular abnormalities in canine mucopolysaccharidosis VII

@article{Sammarco2000EffectsOB,
  title={Effects of bone marrow transplantation on the cardiovascular abnormalities in canine mucopolysaccharidosis VII},
  author={Carl D Sammarco and Max Harry Weil and Christy Just and S Weimelt and Catherine M Hasson and Thomas M O'Malley and S E Evans and Peihe Wang and M Casal and Jeremy Wolfe and Mark Haskins},
  journal={Bone Marrow Transplantation},
  year={2000},
  volume={25},
  pages={1289-1297}
}
The genetic mucopolysaccharidoses (MPS) are a family of lysosomal storage diseases resulting from defective catabolism of glycosaminoglycans (GAGs). Echocardiographic abnormalities in dogs with MPS type VII (Sly syndrome, β-glucuronidase deficiency) included mitral valve thickening and insufficiency, large aortic dimensions in both the long and short axes, and thickened aortic valves. Grossly, at post mortem examination, there was nodular thickening of the mitral valve, a prominent ductus… CONTINUE READING

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Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs.

Proceedings of the National Academy of Sciences of the United States of America • 2002

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