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Updated: Jan 23, 2026

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治疗性抑制Tubb4a可以挽救H-ABC白血病

Sunetra Sase1, Julia L Hacker1, Prabhat R Napit1

  • 1Division of Neurology, Department of Pediatrics, the Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

Molecular therapy : the journal of the American Society of Gene Therapy
|January 22, 2026
PubMed
概括

基底和小脑的低髓化和缩 (H-ABC) 是一种罕见的白血病. 针对TUBB4A的反感性寡核酸 (ASO) 疗法在小鼠模型中显示出延长寿命和改善运动功能的前景.

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