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Batten's disease: failure of allogeneic bone marrow transplantation to arrest disease progression in a canine model
Author(s) -
Deeg H. Joachim,
Shulman Howard M.,
Albrechtsen Dagfin,
Graham Ted C.,
Storb Rainer,
Koppang Nils
Publication year - 1990
Publication title -
clinical genetics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.543
H-Index - 102
eISSN - 1399-0004
pISSN - 0009-9163
DOI - 10.1111/j.1399-0004.1990.tb04188.x
Subject(s) - batten disease , bone marrow , medicine , neuronal ceroid lipofuscinosis , transplantation , total body irradiation , disease , pathology , atrophy , bone marrow transplantation , lysosomal storage disease , cyclophosphamide , chemotherapy
We investigated whether the course of canine ceroid lipofuscinosis (CCL), a model of Batten's disease in man, was affected by allogeneic bone marrow transplantation. Four English setters with CCL, 4 months of age, were given 9.2 Gy of total body irradiation, followed by the infusion of bone marrow cells from healthy DLA identical sibling donors. All transplanted dogs had complete hematologic reconstitution. However, at 12–13 months posttransplant, all dogs developed characteristic and progressive signs of CCL. Autopsies revealed cerebral atrophy and findings of ceroid storage not different from those in non‐transplanted controls. These findings suggest that bone marrow cells do not contain or release the gene product(s) necessary to correct the disease. It appears unlikely that with our current knowledge, allogeneic marrow transplantation would be beneficial in the treatment of Batten's disease.