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

• मुख्य लेखकों: Ming Jin (लेखक), Jiajia Lin (लेखक), Haisen Li (लेखक), Zhifang Li (लेखक), Dong Yang (लेखक), Yin Wang (लेखक), Yuyang Yu (लेखक), Zhurui Shao (लेखक), Long Chen (लेखक), Zhiqiang Wang (लेखक), Yu Zhang (लेखक), Xiumei Zhang (लेखक), Ning Wang (लेखक), Chunlong Xu (लेखक), Hui Yang (लेखक), Wan-Jin Chen (लेखक), Guoling Li (लेखक)
• स्वरूप: पुस्तक
• प्रकाशित: Elsevier, 2024-06-01T00:00:00Z.
• विषय: article
• ऑनलाइन पहुंच: 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...