Adeno-Associated Virus-Mediated RNAi against Mutant Alleles Attenuates Abnormal Calvarial Phenotypes in an Apert Syndrome Mouse Model

Apert syndrome (AS), the most severe form of craniosynostosis, is caused by missense mutations including Pro253Arg(P253R) of fibroblast growth factor receptor 2 (FGFR2), which leads to enhanced FGF/FGFR2-signaling activity. Surgical correction of the deformed skull is the typical treatment for AS. B...

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Vydáno v:Mol Ther Nucleic Acids
Hlavní autoři: Luo, Fengtao, Xie, Yangli, Wang, Zuqiang, Huang, Junlan, Tan, Qiaoyan, Sun, Xianding, Li, Fangfang, Li, Can, Liu, Mi, Zhang, Dali, Xu, Meng, Su, Nan, Ni, Zhenhong, Jiang, Wanling, Chang, Jinhong, Chen, Hangang, Chen, Shuai, Xu, Xiaoling, Deng, Chuxia, Wang, Zhugang, Du, Xiaolan, Chen, Lin
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2018
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Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6197781/
https://ncbi.nlm.nih.gov/pubmed/30321816
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2018.09.012
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