Cationic Nanoemulsions as a Gene Delivery System: Proof of Concept in the Mucopolysaccharidosis I Murine Model.

@article{Fraga2015CationicNA,
  title={Cationic Nanoemulsions as a Gene Delivery System: Proof of Concept in the Mucopolysaccharidosis I Murine Model.},
  author={Michelle Fraga and Talita Giacomet de Carvalho and Dirnete da Silva Diel and N{\'e}lson Alexandre Kretzmann Filho and Helder F Teixeira and Ursula Matte},
  journal={Journal of nanoscience and nanotechnology},
  year={2015},
  volume={15 1},
  pages={810-6}
}
Mucopolysaccharidosis I (MPS I) is an autosomal recessive lysosomal storage disease due to deficient a-L-iduronidase (IDUA) activity. It results in the accumulation of the glycosaminoglycans (GAGs) heparan and dermatan sulfate and leads to several clinical manifestations. This study describes the use of cationic nanoemulsions as a non-viral carrier for the plasmid named pIDUA, which has the gene that encodes for the IDUA enzyme. Cationic nanoemulsions, composed by a medium chain triglycerides… CONTINUE READING