Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
US11767538B2 · kind B2 · utility
Assignees
Inventor
Key dates
| Filing date | Aug 9, 2019 |
| Grant date | Sep 26, 2023 |
| Priority date | — |
| Expiry date | Aug 9, 2039 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2830/008
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present invention relates to compositions and methods, in particular to methods based on systemic injection of rAAV, for delivering genes to cells of the central nervous system in mammals, such as brain neurons or glial cells, and in particular to motor neurons or glial cells of the spinal cord The invention also relates to methods of treating motor neuron disorders in mammals by expression of therapeutic genes. The invention stems from the unexpected discovery that peripheral injection of AAV vectors leads to a bypass of the blood brain barrier and a massive infection of motor neurons. The invention may be used in any mammal, including human subjects.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.