新規血清型に由来するAAVベクターの有用性

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  • 水上 浩明
    自治医科大学分子病態治療研究センター遺伝子治療研究部
  • 小澤 敬也
    自治医科大学分子病態治療研究センター遺伝子治療研究部

書誌事項

タイトル別名
  • Utility of AAV Vectors Derived from Novel Serotypes
  • シンキ ケッセイガタ ニ ユライ スル AAV ベクター ノ ユウヨウセイ

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説明

  AAV vector is derived from nonpathogenic virus and has a number of attractive features as a vector for human gene transfer including safety, broad tissue specificity, and low immunogenicity following gene transfer. Moreover, persistent transgene expression (for years) was demonstrated in multiple animal experiments. For these reasons, applications to a wide spectrum of diseases are expected, and several clinical trials have been conducted. Although it is too early to conclude the outcome, the efficacy of treatment was not sufficiently substantiated in most of the trials despite confirming the safety of the vector. These results are primarily due to low levels of transgene expression. One of the approaches to improve this situation is the use of alternative serotypes of AAV. Traditionally, serotype 2 was considered to be a prototype of AAV, and the majority of studies including human clinical trials have been conducted using this serotype. On the other hand, there are five “classical” serotypes, and several have been additionally discovered from tissues of primates including humans. These serotypes are considered to be valuable resources for vector development to overcome the shortcomings of serotype 2. This review focuses on the difference in expression levels and tissue specificity of various serotype-derived vectors and summarizes current status in the treatment of candidate diseases.<br>

収録刊行物

  • 薬学雑誌

    薬学雑誌 126 (11), 1021-1028, 2006-11-01

    公益社団法人 日本薬学会

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