1篇
您的检索式：作者名="Zhurui Shao"
|
|
|
题名
|
作者
|
年代
|
出处
|
被引量
|
| 1 | enOsCas12f1-mediated exon skipping for Duchenne muscular dystrophy therapy in humanized mouse model显示文摘Duchenne muscular dystrophy(DMD)is a severe neuromuscular disorder caused by mutations in the dystrophin gene,which encodes the essential protein dystrophin.This genetic condition,affecting approximately 1 in 5000 male births worldwide(Birnkrant et al.,2018),is characterized by progressive muscle weakness and wasting,leading to premature death in the second or third decade of life(Farini et al.,2016).The absence of dystrophin protein causes instability in the sarcolemma,which predisposes individuals to myonecrosis and activation of inflammatory signaling cascades.Unfortunately,there is currently no effective cure for DMD,and glucocorticoid steroids are commonly used in clinical settings to delay symptom development(Merlini et al.,2003). | Ming Jin Jiajia Lin Yu Zhang Qingquan Xiao Xiangfeng Kong Xiumei Zhang Zhurui Shao Yin Wang Yuyang Yu Jinjing Li Wan-Jin Chen Guoling Li Hui Yang Ning Wang | 2024 | Journal of Genetics and Genomics2024，51，2： | 0 |
您所在的机构暂未开通此部分的功能，请联系管理员