Gene therapy using human FMRP isoforms driven by the human FMR1 promoter rescues fragile X syndrome mouse deficits

Fragile X syndrome (FXS) is caused by the loss of the fragile X messenger ribonucleoprotein 1 (FMRP) encoded by the FMR1 gene. Gene therapy using adeno-associated virus (AAV) to restore FMRP expression is a promising therapeutic strategy. However, so far AAV gene therapy tests for FXS only utilized...

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Detalles Bibliográficos
Autores principales: Jiang, Yiru, Han, Linkun, Meng, Jian, Wang, Zijie, Zhou, Yunqiang, Yuan, Huilong, Xu, Hui, Zhang, Xian, Zhao, Yingjun, Lu, Jinsheng, Xu, Huaxi, Zhang, Chen, Zhang, Yun-wu
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9593309/
https://www.ncbi.nlm.nih.gov/pubmed/36320413
http://dx.doi.org/10.1016/j.omtm.2022.10.002

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9593309/
https://www.ncbi.nlm.nih.gov/pubmed/36320413
http://dx.doi.org/10.1016/j.omtm.2022.10.002

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