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

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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Bibliographic Details
Main Authors: Xiao Tian, Qingyun Zheng, Jinyan Xie, Qinlinglan Zhou, Letong Liang, Guotong Xu, Hongyan Chen, Chen Ling, Daru Lu
Format: Article
Language:English
Published: Elsevier 2023-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Delivery Strategies
retinitis pigmentosa
rAAV
MFRP
microRNA
gene therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S216225312300118X

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http://www.sciencedirect.com/science/article/pii/S216225312300118X

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