Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres
نویسندگان
چکیده
منابع مشابه
Bone marrow transplantation for feline mucopolysaccharidosis I.
Severe mucopolysaccharidosis type I (MPS I) is a fatal neuropathic lysosomal storage disorder with significant skeletal involvement. Treatment involves bone marrow transplantation (BMT), and although effective, is suboptimal, due to treatment sequelae and residual disease. Improved approaches will need to be tested in animal models and compared to BMT. Herein we report on bone marrow transplant...
متن کاملNeonatal bone marrow transplantation prevents bone pathology in a mouse model of mucopolysaccharidosis type I.
Neonatal bone marrow transplantation (BMT) could offer a novel therapeutic opportunity for genetic disorders by providing sustainable levels of the missing protein at birth, thus preventing tissue damage. We tested this concept in mucopolysaccharidosis type I (MPS IH; Hurler syndrome), a lysosomal storage disorder caused by deficiency of α-l-iduronidase. MPS IH is characterized by a broad spect...
متن کاملBehavioral consequences of bone marrow transplantation in the treatment of murine mucopolysaccharidosis type VII.
The gusmps/gusmps mouse is a model of the human lysosomal storage disease mucopolysaccharidosis type VII caused by deficient beta-glucuronidase activity. Bone marrow transplantation has been shown to correct some of their biochemical and pathological abnormalities but its efficacy in correcting their neurological functional deficits is unknown. We transplanted the neonatal gusmps/gusmps mice an...
متن کاملIncreased Longevity and Metabolic Correction Following Syngeneic Bone Marrow Transplantation in a Murine Model of Mucopolysaccharidosis Type I
Mucopolysaccharidosis type I (MPS I) is an autosomal recessive inherited disease caused by deficiency of the glycosidase α-L-iduronidase (IDUA). Deficiency of IDUA leads to lysosomal accumulation of the glycosaminoglycans (GAG) heparan and dermatan sulfate and associated multi-systemic disease, the most severe form known as Hurler syndrome. Since 1981, the treatment of Hurler patients has often...
متن کاملSyngeneic 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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ژورنال
عنوان ژورنال: Archives of Disease in Childhood
سال: 1997
ISSN: 0003-9888,1468-2044
DOI: 10.1136/adc.76.2.92