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Updated: Jan 21, 2026

A Simple and Low-cost Assay for Measuring Ambulation in Mouse Models of Muscular Dystrophy
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A Simple and Low-cost Assay for Measuring Ambulation in Mouse Models of Muscular Dystrophy

Published on: December 29, 2017

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修飾遺伝子のアップレギュレーションによる筋肉縮に対する変異独立アプローチ

Dwi U Kemaladewi1,2, Prabhpreet S Bassi1,3, Steven Erwood1,3

  • 1Program in Genetics and Genome Biology, the Hospital for Sick Children Research Institute, Toronto, Ontario, Canada.

Nature
|July 26, 2019
PubMed
まとめ

この研究は,先天性筋ジストロフィー1A型 (MDC1A) を修正できる遺伝子であるLama1を上調する新しいCRISPR活性化戦略を示しています. MDC1Aやその他の遺伝的疾患の治療には この変異独立アプローチが有望である.

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