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Improved gene therapy for MFRP deficiency-mediated retinal degeneration by knocking down endogenous bicistronic Mfrp and Ctrp5 transcript

The membrane frizzled-related protein (Mfrp) and C1-tumor necrosis factor related protein 5 (Ctrp5) genes are transcribed as a bicistronic unit and dysregulation of either gene is associated with retinal degeneration in the retinal pigment epithelium (RPE) cells. However, the mechanisms that regulat...

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Detalles Bibliográficos
Autores principales: Tian, Xiao, Zheng, Qingyun, Xie, Jinyan, Zhou, Qinlinglan, Liang, Letong, Xu, Guotong, Chen, Hongyan, Ling, Chen, Lu, Daru
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10238587/
https://www.ncbi.nlm.nih.gov/pubmed/37273779
http://dx.doi.org/10.1016/j.omtn.2023.05.001