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.
Bibliographical noteFunding 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).
- Adeno-associated virus
- Blood-brain barrier
- Intranasal delivery
- Neurological disease