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Duchenne muscular dystrophy treatment with lentiviral vector containing mini‐dystrophin gene in vivo

Abstract Duchenne muscular dystrophy (DMD) is an incurable X‐linked recessive genetic disease caused by mutations in the dystrophin gene. Many researchers aim to restore truncated dystrophin via viral vectors. However, the low packaging capacity and immunogenicity of vectors have hampered their clin...

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Hlavní autoři: Xiaoyu Wang, Yanghui Zhu, Taiqing Liu, Lingyan Zhou, Yunhai Fu, Jinhua Zhao, Yinqi Li, Yeteng Zheng, Xiaodong Yang, Xiangjie Di, Yang Yang, Zhiyao He
Médium: Artigo
Jazyk:Inglês
Vydáno: Wiley 2024-01-01
Edice:MedComm
Témata:
Duchenne muscular dystrophy
dystrophin
gene delivery
gene therapy
lentiviral vector
On-line přístup:https://doi.org/10.1002/mco2.423
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