Author

Hyuk-Jin Cha

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Open access Oct 2025

Mutation-agnostic base editing of the progerin farnesylation site rescues Hutchinson-Gilford progeria syndrome phenotypes in neuromuscular organoids

Hutchinson Gilford progeria syndrome (HGPS) is a fatal premature aging disorder caused by pathogenic farnesylated lamin A variants that disrupt nuclear architecture and DNA repair. Current therapies, including farnesyltransferase inhibitors, provide only modest survival benefits and lack molecular specificity, while mu...

Dong-Woo Kim, Eun-Ji Kwon, Beom-Jin Jeon et al. · bioRxiv · 0 citations

#gene editing Open access Oct 2026

Patient-derived teratomas as a humanized platform for dystrophin restoration by ex vivo cell therapy and in vivo base editing.

Cell and gene therapies hold great promise for treating monogenic disorders, yet their preclinical evaluation remains limited by the lack of scalable, human-specific models. Here we establish a patient-derived teratoma xenograft platform as a proof-of-concept system to evaluate ex vivo and in vivo therapeutic strategie...

Mihn Jeong Park, H. Kim, Yuna Hwang et al. · Experimental and Molecular M... · 0 citations