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Updated: Aug 19, 2026

Modeling Charcot-Marie-Tooth Disease In Vitro by Transfecting Mouse Primary Motoneurons
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Modeling Charcot-Marie-Tooth Disease In Vitro by Transfecting Mouse Primary Motoneurons

Published on: January 7, 2019

ソマティック細胞の遺伝子移植により,マウリン粘膜多糖症第7型における病理の逆転

J H Wolfe1, M S Sands, J E Barker

  • 1Jackson Laboratory, Bar Harbor, Maine 04609.

Nature
|December 24, 1992
PubMed
まとめ

レトロウイルスベクトルを用いた遺伝子治療は,幹細胞における低レベルのβ-グルキュロニダース発現を長期にわたって可能にし,リゾソーム貯蔵を改善することによって,マウスのスライ症候群を部分的に修正します.

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