Mutation-Agnostic Base Editing of the Progerin Farnesylation Site Rescues Hutchinson-Gilford Progeria Syndrome Phenotypes in Neuromuscular Organoids
Dong-Woo Kim, Eun‐Ji Kwon, Beom-Jin Jeon, Dong-Hyeok Kwon, Goo Jang, Youngyoon Yoon, Yuna Hwang, Hyukjin Lee, Hyuk‐Jin Cha
This paper develops FATE, a mutation-agnostic base-editing platform that rescues HGPS phenotypes in neuromuscular organoids by disrupting the LMNA farnesylation motif.
Nature Communications · Aug 31, 2026Peer-reviewedPathology