Clin Genet. 1990 Oct;38(4):274-80.

Bone marrow transplantation in canine GM1 gangliosidosis.

O'Brien JS, Storb R, Raff RF, Harding J, Appelbaum F, Morimoto S, Kishimoto Y, Graham T, Ahern-Rindell A, O'Brien SL.

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Department of Neurosciences, School of Medicine, University of California, San Diego, La Jolla.

Abstract

Allogeneic bone marrow transplantation was carried out in an 81-day-old Portuguese water dog with GM1 gangliosidosis using a DLA identical sibling as donor. Engraftment was complete and beta-galactosidase activity in leukocytes of the transplanted dog were similar to those in the donor. Over the next 2.5 months neurological deterioration in the transplanted dog was similar to that in untreated dogs with GM1 gangliosidosis. Cerebral ganglioside GM1 concentrations were not diminished by bone marrow transplantation and cerebral beta-galactosidase activity was negligible. We conclude that allogeneic bone marrow transplantation early in life is ineffective in canine GM1 gangliosidosis.

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Neuronal-visceral GM1 gangliosidosis in a dog with beta-galactosidase deficiency. [Science. 1976]
Neuronal-visceral GM1 gangliosidosis in a dog with beta-galactosidase deficiency.
Read DH, Harrington DD, Keenana TW, Hinsman EJ. Science. 1976 Oct 22; 194(4263):442-5.
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