sqc (2025-09-01 00:11):
# Paper A Potent Epigenetic Editor Targeting Human PCSK9 for Durable Reduction of Low-Density Lipoprotein Cholesterol Levels, Nature Medicine, Volume 31, April 2025, [DOI](https://doi.org/10.1038/s41591-025-03508-x) 本研究开发了一种针对人类PCSK9基因的表观遗传编辑器，通过诱导DNA甲基化实现LDL-C水平的持久降低。该编辑器在小鼠模型中表现出近乎完全的PCSK9基因沉默，效果持续至少1年，并在部分肝切除诱导的肝脏再生后仍能维持。此外，研究还展示了其可逆性，通过去甲基化激活剂可重新激活PCSK9基因表达。在食蟹猴中，单次给药显著降低了PCSK9蛋白水平（约90%）和LDL-C水平（约70%）。该研究不仅证明了表观遗传编辑在体内治疗应用中的潜力，还支持了开发基于表观遗传编辑器的高胆固醇血症治疗方案。
Nature Medicine, 2025-4. DOI: 10.1038/s41591-025-03508-x
A potent epigenetic editor targeting human PCSK9 for durable reduction of low-density lipoprotein cholesterol levels
Frederic Tremblay, Qiang Xiong, Shrijal S. Shah, Chih-Wei Ko, Kenneth Kelly, Mary S. Morrison, Cristiana Giancarlo, Ricardo N. Ramirez, Erica M. Hildebrand, Sarah B. Voytek, ... >>>
Frederic Tremblay, Qiang Xiong, Shrijal S. Shah, Chih-Wei Ko, Kenneth Kelly, Mary S. Morrison, Cristiana Giancarlo, Ricardo N. Ramirez, Erica M. Hildebrand, Sarah B. Voytek, Gabriel K. El Sebae, Shane H. Wright, Liam Lofgren, Scott Clarkson, Christine Waters, Samantha J. Linder, Songlei Liu, Taesun Eom, Shefal Parikh, Yuki Weber, Salette Martinez, Padma Malyala, Sahar Abubucker, Ari E. Friedland, Morgan L. Maeder, Angelo Lombardo, Vic E. Myer, Aron B. Jaffe <<<
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