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Development of an in Vitro Biopotency Assay for an AAV8 Hemophilia B Gene Therapy Vector Suitable for Clinical Product Release

Johannes Lengler,Sogue Coulibaly,Bernadette Gruber,Reinhard Ilk,Josef Mayrhofer,Friedrich Scheiflinger,Werner Hoellriegl,Falko G. Falkner,Hanspeter Rottensteiner

Molecular Therapy — Methods & Clinical Development（2020）

引用 9|浏览28
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关键词
adeno-associated virus,gene therapy,biopotency,coagulation factor IX
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