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
Artículo
en Inglés
| 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:
Disponible
Índice:
LILACS (Américas)
Asunto principal:
Mucopolisacaridosis I
/
Técnicas de Transferencia de Gen
/
Fibroblastos
/
Vectores Genéticos
/
Iduronidasa
Límite:
Humanos
Idioma:
Inglés
Revista:
Clin. biomed. res
Asunto de la revista:
Medicina
Año:
2017
Tipo del documento:
Artículo
País de afiliación:
Brasil
Institución/País de afiliación:
Hospital de Clínicas de Porto Alegre/BR
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