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Updated: Jul 3, 2026

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针对弗里德里希动脉的抗基因寡核酸扩大GAA⋅TTC重复增加Frataxin表达

Negin Mozafari1,2, Salomé Milagres1,2, Tea Umek1,2,3

  • 1Department of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8, SE-141 52 Huddinge, Stockholm, Sweden.

Molecular therapy. Nucleic acids
|June 9, 2025
PubMed
概括

向GAA⋅TTC重复的寡核酸在弗里德里希的性衰竭细胞中增加了弗拉塔克辛水平. 这种方法表明,通过调高基因表达来治疗这种进展性神经退行性疾病是有前途的.

关键词:
针对DNA的向定位弗里德里希的过敏症是什么这就是H-DNA.亨廷顿氏病是亨廷顿氏病的一种疾病.MT:寡核酸:治疗方法和应用.抗基因是一种抗基因.弗拉塔克辛 (frataxin) 是一种这是一种寡核化物.串联重复重复的重复优化治疗性寡核酸的优化三核酸重复扩张的重复扩张

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