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Updated: Jan 20, 2026

Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy
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基于PPMO的外跳转疗法改善了杜申尼肌肉发育不良的小鼠模型中的呼吸功能

Debolina D Biswas1, Maran Y Hernandez Rodriguez1, Lea El Haddad1

  • 1Division of Pulmonary and Sleep Medicine, Department of Pediatrics, Duke University Medical Center, Box 2644, Durham, NC 27710, USA.

Molecular therapy. Nucleic acids
|January 19, 2026
PubMed
概括

细胞透联的二胺酸 (PPMOs) 在杜氏肌肉发育不良 (DMD) 模型中改善呼吸功能. 这种疗法可以恢复双,减少呼吸道肌肉病理,并为DMD患者提供有前途的治疗方法.

关键词:
杜恩肌肉发育不良症 杜恩肌肉发育不良迪斯特罗芬 (Dystrophin) 是一种消极蛋白质.这是FlexiVent的灵活性.MT:寡核酸:治疗方法和应用.这是一个PPMOPPMO.呼吸 呼吸 呼吸 呼吸异常跳过治疗治疗的治疗方法.强迫振荡计强制振荡计酸二胺酸与酸相结合的 morfolino 寡合物.呼吸系统功能障碍 呼吸系统功能障碍整个身体的整体系统图 (plethysmography).

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