CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE;GENETHON
发明人:
BARKATS, MARTINE
申请号：
CA2701561
公开号：
CA2701561C
申请日:
2008.10.03
申请国别(地区):
CA
年份:
2018
代理人:
摘要：
The present invention relates to compositions and methods, in particular tomethods based on systemic injection ofrAAV, for delivering genes to cells of the central nervous system in mammals,such as brain neurons or glial cells, and in particular tomotor neurons or glial cells of the spinal cord The invention also relates tomethods of treating motor neuron disorders in mammalsby expression of therapeutic genes. The invention stems from the unexpecteddiscovery that peripheral injection of AAV vectorsleads to a bypass of the blood brain barrier and a massive infection of motorneurons. The invention may be used in any mammal,including human subjects.
