Lysosomal enzyme can bypass the blood-brain barrier and reach the CNS following intranasal administration Journal Article uri icon
  • Here we provide the first evidence that therapeutic levels of a lysosomal enzyme can bypass the blood-brain barrier following intranasal administration. alpha-L-iduronidase (IDUA) activity was detected throughout the brains of IDUA-deficient mice following a single intranasal treatment with concentrated Aldurazyme(R) (laronidase) and was also detected after intranasal treatment with an adeno-associated virus (AAV) vector expressing human IDUA. These results suggest that intranasal routes of delivery may be efficacious in the treatment of lysosomal storage disorders.

  • Link to Article
    publication date
  • 2012
  • published in
  • Animal Studies
  • Blood
  • Brain
  • Drugs and Drug Therapy
  • Genetics
  • Intranasal Administration
  • Additional Document Info
  • 106
  • issue
  • 1