Delivery of MECP2 polynucleotide using recombinant AAV9
US9415121B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Mar 14, 2013 |
| Grant date | Aug 16, 2016 |
| Priority date | — |
| Expiry date | Mar 14, 2033 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2830/008
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present invention relates to Adeno-associated virus 9 methods and materials useful for systemically delivering polynucleotides across the blood brain barrier. Accordingly, the present invention also relates to methods and materials useful for systemically delivering polynucleotides to the central and peripheral nervous systems. The present invention also relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as spinal muscle atrophy and amyotrophic lateral sclerosis as well as Pompe disease and lysosomal storage disorders. Use of the methods and materials is also indicated, for example, for treatment of Rett syndrome.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.