Title:
ＡＡＶベクターの末梢注入を使用する広範囲におよぶ運動ニューロンへの遺伝子の送達
Document Type and Number:
Japanese Patent JP5620819
Kind Code:
B2
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.
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Inventors:
バルカ，マルティーヌ
Application Number:
JP2010527467A
Publication Date:
November 05, 2014
Filing Date:
October 03, 2008
Export Citation:
Assignee:
ジェネトン
サントル・ナショナル・ドゥ・ラ・ルシェルシュ・シャンティフィクＣＥＮＴＲＥ ＮＡＴＩＯＮＡＬ ＤＥ ＬＡＲＥＣＨＥＲＣＨＥ ＳＣＩＥＮＴＩＦＩＱＵＥ
サントル・ナショナル・ドゥ・ラ・ルシェルシュ・シャンティフィクＣＥＮＴＲＥ ＮＡＴＩＯＮＡＬ ＤＥ ＬＡＲＥＣＨＥＲＣＨＥ ＳＣＩＥＮＴＩＦＩＱＵＥ
International Classes:
A61K31/7105; A61K9/08; A61K47/30; A61K48/00; A61P17/02; A61P21/00; A61P25/00; A61P25/20; A61P29/00; A61P35/00; A61P37/06; A61P43/00
Attorney, Agent or Firm:
Hajime Tsukuni
Saito a fringe -- happy
Saito a fringe -- happy