Adeno-associated virus gene transfer in Morquio A disease - effect of promoters and sulfatase-modifying factor 1.

@article{AlmcigaDaz2010AdenoassociatedVG,
  title={Adeno-associated virus gene transfer in Morquio A disease - effect of promoters and sulfatase-modifying factor 1.},
  author={Carlos J Alm{\'e}ciga-D{\'i}az and Adriana Maria Monta{\~n}o and Shunji Tomatsu and L. A. Barrera},
  journal={The FEBS journal},
  year={2010},
  volume={277 17},
  pages={3608-19}
}
Mucopolysaccharidosis (MPS) IVA is an autosomal recessive disorder caused by deficiency of the lysosomal enzyme N-acetylgalatosamine-6-sulfate sulfatase (GALNS), which leads to the accumulation of keratan sulfate and chondroitin 6-sulfate, mainly in bone. To explore the possibility of gene therapy for Morquio A disease, we transduced the GALNS gene into HEK293 cells, human MPS IVA fibroblasts and murine MPS IVA chondrocytes by using adeno-associated virus (AAV)-based vectors, which carry human… CONTINUE READING
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