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

• Main Authors: Xiao Tian (Author), Qingyun Zheng (Author), Jinyan Xie (Author), Qinlinglan Zhou (Author), Letong Liang (Author), Guotong Xu (Author), Hongyan Chen (Author), Chen Ling (Author), Daru Lu (Author)
• Format: Book
• Published: Elsevier, 2023-06-01T00:00:00Z.
• Subjects: article
• Online Access: 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...