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Updated: Mar 30, 2026

CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors
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ジャグド1はダッチェンヌ筋縮症のフェノタイプを救う

Natassia M Vieira1, Ingegerd Elvers2, Matthew S Alexander3

  • 1The Division of Genetics and Genomics, Boston Children's Hospital, Boston, MA 02115, USA; Department of Pediatrics and Genetics, Harvard Medical School, Boston, MA 02115, USA; Human Genome and Stem Cell Center, Biosciences Institute, University of São Paulo, São Paulo 05508-090, Brazil.

Cell
|November 20, 2015
PubMed
まとめ

軽度のゴールデン・レトリバーの筋肉縮症の犬は,ジャグド1遺伝子発現が増加している. これは,ジャガド1がダッチェンヌ筋縮症の治療標的であり,ディストロフィンに独立した治療アプローチを提供することを示唆しています.

キーワード:
DMD についてジャグされた1ディストロフィン遺伝子組み換え筋肉

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