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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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6197781/
https://www.ncbi.nlm.nih.gov/pubmed/30321816
http://dx.doi.org/10.1016/j.omtn.2018.09.012

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