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Sanfilippo disease Supported projects Bone marrow transplant

Bone marrow transplant

(Research work by Dr Adeline Lau, University of Adelaide, Australia)

Studies carried out by Dr Adeline Lau at the Women’s and Children’s Hospital, Adelaide, Australia, have shown that bone marrow transplantation from healthy mice to baby mice which in time will develop Sanfilippo disease, do not prevent or delay the apparition of neurological lesions. Although bone marrow transplantation are used with success in several lysosomal storage disorders, the study concluded that this approach does not constitute a clinically useful approach for MPS III.