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    • 3. 发明申请
    • ADENO-ASSOCIATED VIRUS VARIANTS AND METHODS OF USE THEREOF
    • ADENO相关病毒变种及其使用方法
    • US20160017295A1
    • 2016-01-21
    • US14774972
    • 2014-05-29
    • THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
    • David V. SchafferMelissa A. KottermanBum-Yeol HwangJames T. Koerber
    • C12N7/00C12N15/10A61K35/76A61K48/00C07K14/005
    • The present disclosure provides infectious recombinant adeno-associated virus (rAAV) virions that comprise a variant capsid protein and a heterologous nucleic acid. The present disclosure further provides the variant adeno-associated virus (AAV) capsid proteins (and/or a nucleic acid encoding the variant AAV capsid proteins), which confer to an infectious rAAV virion an increased resistance to human AAV neutralizing antibodies. The present disclosure further provides host cells comprising an infectious rAAV virion and/or a nucleic acid encoding a subject variant AAV capsid protein. The present disclosure further provides methods of delivering a heterologous nucleic acid to a target cell where the target cell is contacted with a subject infectious rAAV virion. The present disclosure 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.
    • 本公开提供包含变体衣壳蛋白和异源核酸的感染性重组腺相关病毒(rAAV)病毒粒子。 本公开进一步提供变体腺相关病毒(AAV)衣壳蛋白(和/或编码变体AAV衣壳蛋白的核酸),其赋予感染性rAAV病毒粒子增加的对人AAV中和抗体的抗性。 本发明进一步提供包含感染性rAAV病毒粒子和/或编码主题变体AAV衣壳蛋白的核酸的宿主细胞。 本公开进一步提供将异源核酸递送至靶细胞的方法,其中靶细胞与受试者感染性rAAV病毒粒子接触。 本公开进一步提供将个体基因产物递送给个体的方法,所述方法通常涉及向有需要的个体施用有效量的受试者rAAV病毒粒子。