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

• Автори: Xiao Tian (Автор), Qingyun Zheng (Автор), Jinyan Xie (Автор), Qinlinglan Zhou (Автор), Letong Liang (Автор), Guotong Xu (Автор), Hongyan Chen (Автор), Chen Ling (Автор), Daru Lu (Автор)
• Формат: Книга
• Опубліковано: Elsevier, 2023-06-01T00:00:00Z.
• Предмети: article
• Онлайн доступ: 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...