| First Author | Wolf DA | Year | 2012 |
| Journal | Mol Genet Metab | Volume | 106 |
| Issue | 1 | Pages | 131-4 |
| PubMed ID | 22420937 | Mgi Jnum | J:183240 |
| Mgi Id | MGI:5318123 | Doi | 10.1016/j.ymgme.2012.02.006 |
| Citation | Wolf DA, et al. (2012) Lysosomal enzyme can bypass the blood-brain barrier and reach the CNS following intranasal administration. Mol Genet Metab 106(1):131-4 |
| abstractText | 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. |
