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| 1. | Title: Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice
Author(s): McCarty DM, DiRosario J, Gulaid K, et al. Source: GENE THERAPY Volume: 16 Issue: 11 Pages: 1340-1352 Published: NOV 2009 |
| 2. | Title: Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy
Author(s): Chen YH, Chang M, Davidson BL Source: NATURE MEDICINE Volume: 15 Issue: 10 Pages: 1215-U145 Published: OCT 2009 |
| 3. | Title: Controlling brain tumor growth by intraventricular administration of an AAV vector encoding IFN-beta
Author(s): Meijer DH, Maguire CA, LeRoy SG, et al. Source: CANCER GENE THERAPY Volume: 16 Issue: 8 Pages: 664-671 Published: AUG 2009 |
| 4. | Title: Human alpha-Iduronidase Gene Transfer Mediated by Adeno-Associated Virus Types 1, 2, and 5 in the Brain of Nonhuman Primates: Vector Diffusion and Biodistribution
Author(s): Ciron C, Cressant A, Roux F, et al. Source: HUMAN GENE THERAPY Volume: 20 Issue: 4 Pages: 350-360 Published: APR 2009 |
| 5. | Title: Mechanisms of distribution of mouse beta-galactosidase in the adult GM1-gangliosidosis brain
Author(s): Broekman MLD, Tierney LA, Benn C, et al. Source: GENE THERAPY Volume: 16 Issue: 2 Pages: 303-308 Published: FEB 2009 |
| 6. | Title: Recent developments in adeno-associated virus vector technology
Author(s): Buning H, Perabo L, Coutelle O, et al. Source: JOURNAL OF GENE MEDICINE Volume: 10 Issue: 7 Pages: 717-733 Published: JUL 2008 |
| 7. | Title: Lysosomal enzyme replacement of the brain with intravenous non-viral gene transfer
Author(s): Zhang Y, Wang Y, Boado RJ, et al. Source: PHARMACEUTICAL RESEARCH Volume: 25 Issue: 2 Pages: 400-406 Published: FEB 2008 |
| 8. | Title: Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis
Author(s): Chang M, Cooper JD, Sleat DE, et al. Source: MOLECULAR THERAPY Volume: 16 Issue: 4 Pages: 649-656 Published: APR 2008 |
| 9. | Title: CNS-directed gene therapy for lysosomal storage diseases
Author(s): Sands MS, Haskins ME Source: ACTA PAEDIATRICA Volume: 97 Pages: 22-27 Supplement: Suppl. 457 Published: APR 2008 |
| 10. | Title: Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes
Author(s): Fraldi A, Hemsley K, Crawley A, et al. Source: HUMAN MOLECULAR GENETICS Volume: 16 Issue: 22 Pages: 2693-2702 Published: NOV 15 2007 |