专利内容由知识产权出版社提供
专利名称：Methods for increasing the efficiency of
recombinant AAV product
发明人：Richard Jude Samulski,Xiao Xiao,Richard
Snyder
申请号：US10261188申请日：20020930公开号：US07229823B2公开日：20070612
摘要：The present invention relates to methods and compositions for increasing theproduction of high titre stocks of recombinant AAV (rAAV) through regulation ofexpression of the AAV REP and CAP proteins. The methods and compositions of theinvention are based on the observation that the low level expression of the AAV REPprotein increases the production of AAV viral capsid protein and efficiency of packagingresulting in production of higher titre recombinant viral stocks. The invention
encompasses recombinant AAV vectors that direct the expression of AAV REP and CAPproteins and the use of such vectors for the production of novel stable cell lines capableof generating high titre rAAV vectors. The invention provides methods for regulating theexpression of the AAV REP gene at the transcriptional and post-translational level. Themethods and compositions of the invention can be used to produce high titre stocks ofrAAV which can be used in gene therapy for the purpose of transferring geneticinformation into appropriate host cells for the management and correction of humandiseases including inherited and acquired disorders.
申请人：Richard Jude Samulski,Xiao Xiao,Richard Snyder
地址：102 Darlin Cir. Chapel Hill NC 27514 US,2433 Dogwood Dr. Wexford PA 15090US,3445 Guido St. Oakland CA 94602 US
国籍：US,US,US
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