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Updated: Sep 10, 2025

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
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腺髓神经病的基因疗法:挑战,目标细胞和前景

Pierre Bougnères1,2, Catherine Le Stunff1,3, Romina Aron Badin1

  • 1Laboratoire des Maladies Neurodégénératives, MIRCen Institute, Commissariat à l'Energie Atomique, 92260 Fontenay-aux-Roses, France.

Biomedicines
|August 28, 2025
PubMed
概括

基因疗法对X关联上腺缩症 (X-ALD) 是一种神经退行性疾病具有前景. 使用腺相关病毒载体的小鼠模型研究旨在确定治疗这种罕见遗传疾病的最佳点.

关键词:
一个 AAV上腺神经病变轴心病症基因疗法脊髓

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