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Mol Genet Metab. 2012 May;106(1):131-4. doi: 10.1016/j.ymgme.2012.02.006. Epub 2012 Feb 10.

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

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1
Gene Therapy Program, Institute of Human Genetics, Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis MN 55455, USA.

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.

PMID:
22420937
DOI:
10.1016/j.ymgme.2012.02.006
[Indexed for MEDLINE]
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