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MUTANT ADENO-ASSOCIATED VIRUS VIRIONS AND METHODS OF USE THEREOF

机译:突变体腺相关病毒病毒病毒病毒病毒病毒病毒病毒病毒株及其使用方法

摘要

The present invention provides mutant adeno-associated virus (AAV) that exhibit altered capsid properties, e.g., reduced binding to neutralizing antibodies in serum and/or altered heparin binding and/or altered infectivity of particular cell types. The present invention further provides libraries of mutant AAV comprising one or more mutations in a capsid gene. The present invention further provides methods of generating the mutant AAV and mutant AAV libraries, and compositions comprising the mutant AAV. The present invention further provides recombinant AAV (rAAV) virions that comprise a mutant capsid protein. The present invention further provides nucleic acids comprising nucleotide sequences that encode mutant capsid proteins, and host cells comprising the nucleic acids. The present invention further provides methods of delivering a gene product to an individual, the methods generally involving administering an effective amount of a subject rAAV virion to an individual in need thereof.
机译:本发明提供了突变体腺相关病毒(AAV),其表现出改变的衣壳性质,例如,减少与血清中和抗体中和和/或改变特定细胞类型的肝素结合和/或改变感染性的结合。本发明进一步提供突变AAV文库,其包含衣壳基因中的一种或多种突变。本发明进一步提供了产生突变AAV和突变AAV文库的方法,以及包含突变AAV的组合物。本发明进一步提供包含突变胶囊蛋白的重组AAV(RAAV)病毒粒子。本发明进一步提供了包含编码突变胶囊蛋白的核苷酸序列的核酸,以及包含核酸的宿主细胞。本发明进一步提供将基因产物递送给个体的方法,这些方法通常涉及向有此需要的个体施用有效量的受试者rAAv病毒。

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