INTRATHECAL DELIVERY OF RECOMBINANT ADENO-ASSOCIAT
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专利名称：INTRATHECAL DELIVERY OF RECOMBINANT ADENO-ASSOCIATED VIRUS 9
发明人：KASPAR, Brian, K.,PORENSKY, Paul,BURGHES, Arthur
申请号：EP13747755.0
申请日：20130731
公开号：EP2879719A1
公开日：
20150610
专利内容由知识产权出版社提供
摘要：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. It is disclosed that administration of a non-ionic, low-osmolar contrast agent, together with a rAA9 vector for the expression of Survival Motor Neuron protein, improves the survival of SMN mutant mice as compared to the administration of the expression vector alone.
申请人：Nationwide Children's Hospital,Ohio State Innovation Foundation
地址：700 Children's Hospital Drive Room W172 Columbus, OH 43205 US,1524 North High Street Columbus, OH 43201 US
国籍：US,US
代理机构：Holliday, Louise Caroline
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