Syngeneic Bone Marrow Transplantation Reduces the Hearing Loss Associated With Murine Mucopolysaccharidosis Type
نویسندگان
چکیده
MPS VI1 mice are deficient in P-glucuronidase and share many clinical, biochemical, and pathologic characteristics with human mucopolysaccharidosis type VI1 (MPS VII). We have shown that syngeneic bone marrow transplantation (BMT) prolongs survival and reduces lysosomal storage in many organs of the MPS VI1 mouse. In this report, we quantify the hearing loss and determine the impact of syngeneic BMT on the development of deafness and the associated pathology in the MPS VI1 mouse. Eleven weeks after syngeneic BMT performed at birth, treated MPS VI1 mice had normal audtory-evoked brainstem responses (ABR), whereas untreated MPS VI1 mice had ABR thresholds 43 dB higher
منابع مشابه
Syngeneic bone marrow transplantation reduces the hearing loss associated with murine mucopolysaccharidosis type VII.
MPS VII mice are deficient in beta-glucuronidase and share many clinical, biochemical, and pathologic characteristics with human mucopolysaccharidosis type VII (MPS VII). We have shown that syngeneic bone marrow transplantation (BMT) prolongs survival and reduces lysosomal storage in many organs of the MPS VII mouse. In this report, we quantify the hearing loss and determine the impact of synge...
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متن کاملBehavior and Therapeutic Efficacy of b-Glucuronidase–Positive Mononuclear Phagocytes in a Murine Model of Mucopolysaccharidosis Type VII
Bone marrow transplantation (BMT) is relatively effective for the treatment of lysosomal storage diseases. To better understand the contribution of specific hematopoietic lineages to the efficacy of BMT, we transplanted b-glucuronidase–positive mononuclear phagocytes derived from either the peritoneum or from bone marrow in vitro into syngeneic recipients with mucopolysaccharidosis type VII (MP...
متن کاملBehavior and therapeutic efficacy of beta-glucuronidase-positive mononuclear phagocytes in a murine model of mucopolysaccharidosis type VII.
Bone marrow transplantation (BMT) is relatively effective for the treatment of lysosomal storage diseases. To better understand the contribution of specific hematopoietic lineages to the efficacy of BMT, we transplanted beta-glucuronidase-positive mononuclear phagocytes derived from either the peritoneum or from bone marrow in vitro into syngeneic recipients with mucopolysaccharidosis type VII ...
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