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

• Hoofdauteurs: Xiao Tian (Auteur), Qingyun Zheng (Auteur), Jinyan Xie (Auteur), Qinlinglan Zhou (Auteur), Letong Liang (Auteur), Guotong Xu (Auteur), Hongyan Chen (Auteur), Chen Ling (Auteur), Daru Lu (Auteur)
• Formaat: Boek
• Gepubliceerd in: Elsevier, 2023-06-01T00:00:00Z.
• Onderwerpen: article
• Online toegang: Connect to this object online.

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...