Systematic in vivo candidate evaluation uncovers therapeutic targets for LMNA dilated cardiomyopathy and risk of Lamin A toxicity.

Chia Yee TanPui Shi ChanHansen TanSung Wei TanChang Jie Mick LeeJiong-Wei WangShu YeHendrikje WernerYing Jie LohYin Loon LeeMatthew Ackers-JohnsonRoger S Y FooJianming Jiang

Published in: Journal of translational medicine (2023)

In vivo candidate evaluation revealed that supplementation of Lamin C or knockdown of Sun1 significantly suppressed Lmna DCM and achieve prolonged survival. Conversely, Lamin A supplementation did not rescue long term survival and may impart detrimental cardiotoxicity risk. This study highlights a potential of advancing Lamin C and Sun1 as therapeutic targets for the treatment of LMNA DCM.

Keyphrases

muscular dystrophy
oxidative stress
single cell
climate change
combination therapy
replacement therapy