Improvement in the range of joint motion in seven patients with mucopolysaccharidosis type II during experimental gene expression-targeted isoflavone therapy (GET IT).
Am J Med Genet A
; 155A(9): 2257-62, 2011 Sep.
Article
em En
| MEDLINE
| ID: mdl-21834048
ABSTRACT
Mucopolysaccharidosis type II (MPS II, Hunter disease) is an X chromosome-linked inherited metabolic disease caused by mutations resulting in deficiency of activity of iduronate-2-sulfatase (IDS) and accumulation of undegraded glycosaminoglycans (GAGs), heparan sulfate, and dermatan sulfate. Previous experiments with cell cultures and studies on animal model of MPS II suggested that gene expression-targeted isoflavone therapy (GET IT), based on genistein-mediated reduction of efficiency of GAG synthesis, might be a suitable therapy for this disease. In this report, we demonstrate efficacy of GET IT in connective tissue elasticity, particularly in improving the range of joint motion in seven patients with MPS II after 26 weeks of treatment with an isoflavone extract at the dose corresponding to 5 mg/kg/day of genistein.
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Amplitude de Movimento Articular
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Mucopolissacaridose II
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Tecido Conjuntivo
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Genisteína
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Isoflavonas
Limite:
Adolescent
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Adult
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Child
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Humans
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Male
Idioma:
En
Ano de publicação:
2011
Tipo de documento:
Article