Correction of human nonsense mutation via adenine base editing for Duchenne muscular dystrophy treatment in mouse

• 主要な著者: Ming Jin (著者), Jiajia Lin (著者), Haisen Li (著者), Zhifang Li (著者), Dong Yang (著者), Yin Wang (著者), Yuyang Yu (著者), Zhurui Shao (著者), Long Chen (著者), Zhiqiang Wang (著者), Yu Zhang (著者), Xiumei Zhang (著者), Ning Wang (著者), Chunlong Xu (著者), Hui Yang (著者), Wan-Jin Chen (著者), Guoling Li (著者)
• フォーマット: 図書
• 出版事項: Elsevier, 2024-06-01T00:00:00Z.
• 主題: article
• オンライン･アクセス: Connect to this object online.

Duchenne muscular dystrophy (DMD) is the most prevalent herediatry disease in men, characterized by dystrophin deficiency, progressive muscle wasting, cardiac insufficiency, and premature mortality, with no effective therapeutic options. Here, we investigated whether adenine base editing can correct...