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SERIES OF RECOMBINANT ADENO-ASSOCIATED VIRUS USEFUL FOR INDUCING RNAI PATHWAY AND GENE THERAPY

机译:用于诱导RNAi途径和基因治疗的重组腺伴随病毒

摘要

The invention relates to a series of recombinant adeno-associated virus that mediates RNA interference (RNAi), useful for gene therapying. In particular, the invention relates to a series of recombinant adeno-associated virus that specially inhibits genes associated with particular diseases by mediating RNAi pathway, and therefore are useful for gene therapy, and the strategy and method of constructing it and the use of thereof.
机译:本发明涉及介导RNA干扰(RNAi)的一系列重组腺相关病毒,可用于基因治疗。特别地,本发明涉及一系列重组腺相关病毒,其通过介导RNAi途径特别抑制与特定疾病相关的基因,因此可用于基因治疗,及其构建策略和方法及其用途。

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