AMPK modulation ameliorates dominant disease phenotypes of CTRP5 variant in retinal degeneration

Kiyoharu J. Miyagishima; Ruchi Sharma; Malika Nimmagadda; Katharina Clore-Gronenborn; Zoya Qureshy; Davide Ortolan; Devika Bose; Mitra Farnoodian; Congxiao Zhang; Andrew Fausey; Yuri V. Sergeev; Mones Abu-Asab; Bokkyoo Jun; Khanh V. Do; Marie-Audrey Kautzman Guerin; Jorgelina Calandria; Aman George; Bin Guan; Qin Wan; Rachel C. Sharp; Catherine Cukras; Paul A. Sieving; Robert B. Hufnagel; Nicolas G. Bazan; Kathleen Boesze-Battaglia; Sheldon Miller; Kapil Bharti

AMPK modulation ameliorates dominant disease phenotypes of CTRP5 variant in retinal degeneration

2021

Miyagishima et al. investigate the pathological mechanisms underlying mutant CTRP5 function in late-onset retinal degeneration (L-ORD). With human iPSC-derived RPE cells, they demonstrate that in L-ORD iRPE, constitutive activation of AMPK disrupts cellular metabolism/energy homoeostasis, changes apical/basal VEGF secretion, and Metformin treatment corrects these associated phenotypes.

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