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Title:
組換えアデノ随伴ウイルス9の髄腔内送達
Document Type and Number:
Japanese Patent JP6708686
Kind Code:
B2
Abstract:
The present invention relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as SMA and ALS as well as Pompe disease and lysosomal storage disorders.

Inventors:
Brian K. Caspar
Arthur Burgess
Paul Polensky
Application Number:
JP2018058524A
Publication Date:
June 10, 2020
Filing Date:
March 26, 2018
Export Citation:
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Assignee:
Nationwide Children's Hospital
Ohio State Innovation Foundation
International Classes:
A61K48/00; A61K31/7088; A61K47/16; A61P21/00; A61P25/02
Domestic Patent References:
JP2007528424A
Foreign References:
WO2011133890A1
Other References:
Molecular Therapy,2011年,Vol.19(11),pp.1971-1980
Can. J. Anesth.,2001年,Vol.48(9),pp.890-893
Attorney, Agent or Firm:
Hidesaku Yamamoto
Natsuki Morishita
Takatoshi Iida
Daisuke Ishikawa
Kensaku Yamamoto