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
Article
in English
| 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)
Full text:
Available
Index:
LILACS (Americas)
Main subject:
Mucopolysaccharidosis I
/
Gene Transfer Techniques
/
Fibroblasts
/
Genetic Vectors
/
Iduronidase
Limits:
Humans
Language:
English
Journal:
Clin. biomed. res
Journal subject:
Medicine
Year:
2017
Type:
Article
Affiliation country:
Brazil
Institution/Affiliation country:
Hospital de Clínicas de Porto Alegre/BR
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