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Viral vectors for efficient transgene delivery

机译:病毒载体可高效传递基因

摘要

The invention provides viral vector formulations and methods of uses thereof for delivery of transgenes or therapeutic nucleic acids to human subjects. The formulations include a vector and suitable amounts of empty capsids, viral genome-containing capsids, or viral capsid proteins which are optionally chemically or structurally modified and which bind to neutralizing anti- AAV antibodies thereby reducing or preventing antibody-mediated clearance of the vector, but still allowing the genome-containing (therapeutic) vector to transduce target cells and achieve therapeutic gene transfer.
机译:本发明提供了病毒载体制剂及其用于将转基因或治疗性核酸递送至人类受试者的用途的方法。所述制剂包括载体和适量的空衣壳,含病毒基因组的衣壳或病毒衣壳蛋白,其任选地经过化学或结构修饰,并与中和的抗AAV抗体结合,从而减少或防止抗体介导的载体清除,但仍允许包含基因组的(治疗性)载体转导靶细胞并实现治疗性基因转移。

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