Lysosomal enzyme can bypass the blood-brain barrier and reach the CNS following intranasal administration

Daniel A. Wolf, Leah R. Hanson, Elena L. Aronovich, Zhenhong Nan, Walter C. Low, William H. Frey, R. Scott McIvor

Research output: Contribution to journalArticlepeer-review

34 Scopus citations

Abstract

Here we provide the first evidence that therapeutic levels of a lysosomal enzyme can bypass the blood-brain barrier following intranasal administration α-l-iduronidase (IDUA) activity was detected throughout the brains of IDUA-deficient mice following a single intranasal treatment with concentrated Aldurazyme® (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.

Original languageEnglish (US)
Pages (from-to)131-134
Number of pages4
JournalMolecular Genetics and Metabolism
Volume106
Issue number1
DOIs
StatePublished - May 2012

Bibliographical note

Funding Information:
We would like to thank B. Koniar for excellent animal care. This work was supported by NIH grants P01HD032652 (RSM), 1R01DK082516 (Dr. Perry Hackett), and training grant T32 DA022616-01 (DAW).

Keywords

  • Adeno-associated virus
  • Alpha-l-iduronidase
  • Blood-brain barrier
  • Intranasal delivery
  • Mucopolysaccharidosis
  • Neurological disease

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