Long-term restoration of alpha-L-iduronidase activity in fibroblasts from patients with mucopolysaccharidosis type I after non-viral gene transfer
Clin. biomed. res
;
37(4): 330-333, 2017. ilus, graf
Artigo
em Inglês
| LILACS
| ID: biblio-876699
ABSTRACT
Mucopolysaccharidosis type I (MPS I) is a lysosomal storage disorder caused by deficiency of alpha-L-iduronidase (IDUA). Limitations such as the need for weekly injections, high morbidity and mortality, and high cost of current treatments show that new approaches to treat this disease are required. In this study, we aimed to correct fibroblasts from a patient with MPS I using non-viral gene therapy. Using a plasmid encoding the human IDUA cDNA, we achieved stable high IDUA levels in transfected fibroblasts up to 6 months of treatment. These results serve as proof of concept that a non-viral approach can correct the enzyme deficiency in cells of patients with lysosomal storage disorders, which can be used as a research tool for a series of disease aspects. Future studies should focus on showing if this approach can be useful in small animals and clinical trials (AU)
Texto completo:
DisponíveL
Índice:
LILACS (Américas)
Assunto principal:
Mucopolissacaridose I
/
Técnicas de Transferência de Genes
/
Fibroblastos
/
Vetores Genéticos
/
Iduronidase
Limite:
Humanos
Idioma:
Inglês
Revista:
Clin. biomed. res
Assunto da revista:
Medicina
Ano de publicação:
2017
Tipo de documento:
Artigo
País de afiliação:
Brasil
Instituição/País de afiliação:
Hospital de Clínicas de Porto Alegre/BR
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