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

• Autori principali: Xiao Tian (Autore), Qingyun Zheng (Autore), Jinyan Xie (Autore), Qinlinglan Zhou (Autore), Letong Liang (Autore), Guotong Xu (Autore), Hongyan Chen (Autore), Chen Ling (Autore), Daru Lu (Autore)
• Natura: Libro
• Pubblicazione: Elsevier, 2023-06-01T00:00:00Z.
• Soggetti: article
• Accesso online: 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...