Centre National de la Recherche Scientifique;GENETHON
发明人:
BARKATS, MARTINE
申请号：
PL08836776
公开号：
PL2212424T3
申请日:
2008.10.03
申请国别(地区):
PL
年份:
2014
代理人:
摘要：
The present invention relates to compositions and methods for delivering genes to cells of the central nervous system, such as motor neurons, in mammals. 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.
