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Article

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

9 May 2023

Abstract excerpt

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 regulate the expression of the bicistronic transcript remain controversial. Here, we identified a microRNA-based...

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PMID: 37273779
DOI: 10.1016/j.omtn.2023.05.001

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