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- Title
Uso de vectores derivados de virus adenoasociados para el tratamiento de la enfermedad de Morquio A.
- Authors
ALMÉCIGA-DÍAZ, CARLOS JAVIER; RUEDA-PÁRAMO, MARÍA ANDREA; ECHEVERRI, OLGA YANETH; MONTA˜O, ADRIANA MARÍA; TOMATSU, SHUNJI; BARRERA, LUIS ALEJANDRO
- Abstract
Toward the development of a gene therapy strategy for the mucopolysaccharidosis IV A (MPS IV A, Morquio A ), in this work we evaluated the capability of an AAV vector to express the N-acetilgalactosamine- 6-sulfate sulfatase (GALNS) gene in HEK293 cells, human mucopolysaccharidosis IV A fibroblasts and murine MPS IV A chondrocites. GALNS activity in lysated cells was increased between 5 and 24 folds)lds compared to nontransfected cells. Also, the effect of coexpression with the sultatase modifiying factor I (SUMF1) was evaluated. GALNS activity was increased up to 4.5 folds in lysated cells compared to those values observed in AAV-GALNS transduced cells. Noteworthy, enzyme activity was only detected in culture media when cells were cotransfected with AAV-GALNS and AAV-SUMFI. These results represent a valuable step in the development of a gene therapy strategy for mucopolysaccharidosis IV A using AAV vectors.
- Subjects
GENETIC vectors; ADENOVIRUSES; MUCOPOLYSACCHARIDOSIS; GENE therapy; ENZYME kinetics; CELL culture
- Publication
Universitas Médica, 2009, Vol 50, Issue 3, p356
- ISSN
0041-9095
- Publication type
Article