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ハンチントン病の治療戦略:現在のアプローチと将来の方向

Mehak Gulzar1, Sana Kauser2, Sumaiya Khan1

  • 1Centre for Interdisciplinary Research in Basic Sciences, Jamia Millia Islamia, New Delhi, India.

Neurodegenerative disease management
|August 28, 2025
PubMed
まとめ

ハンチントン病 (HD) の治療は進歩しており,遺伝子治療と小分子による変異ハンチントン (mHTT) レベルの低下に焦点を当てています. よりよい患者治療の成果を出すために,出産と安全性の課題を克服するためにさらなる研究が必要である.

キーワード:
CAG-リピートCRISPRについてFDAが承認した薬ハンティントン病変異したハンティングチンタンパク質神経変性障害

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