Invention Grant
- Patent Title: Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
-
Application No.: US16536503Application Date: 2019-08-09
-
Publication No.: US11767538B2Publication Date: 2023-09-26
- Inventor: Martine Barkats
- Applicant: GENETHON , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE
- Applicant Address: FR Evry
- Assignee: GENETHON,CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE
- Current Assignee: GENETHON,CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE
- Current Assignee Address: FR Evry; FR Paris
- Agency: Arrigo, Lee, Guttman & Mouta-Bellum LLP
- Priority: EP 301435 2007.10.05
- The original application number of the division: US15713347 2017.09.22
- Main IPC: C12N15/86
- IPC: C12N15/86 ; A61K48/00

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.
Public/Granted literature
- US20200010852A1 WIDESPREAD GENE DELIVERY TO MOTOR NEURONS USING PERIPHERAL INJECTION OF AAV VECTORS Public/Granted day:2020-01-09
Information query
IPC分类: