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

作者：Wolf Daniel A; Hanson Leah R; Aronovich Elena L; Nan Zhenhong; Low Walter C; Frey William H II; McIvor R Scott^*

来源：Molecular Genetics and Metabolism, 2012, 106(1): 131-134.

DOI：10.1016/j.ymgme.2012.02.006

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

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.

• 出版日期2012-5