Recapitulating familial hypercholesterolemia in a mouse model by knock‐in patient‐specific LDLR mutation

Familial hypercholesterolemia (FH) is one of the most prevalent monogenetic disorders leading to cardiovascular disease (CVD) worldwide. Mutations in Ldlr, encoding a membrane‐spanning protein, account for the majority of FH cases. No effective and safe clinical treatments are available for FH. Aden...

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Veröffentlicht in:	The FASEB journal 2024-03, Vol.38 (6), p.e23573-n/a
Hauptverfasser:	Liu, Jing, Yang, Fayu, Shang, Lu, Cai, Shuo, Wu, Yuting, Liu, Yingchun, Zhang, Lifang, Fei, Chenzhong, Wang, Mi, Gu, Feng
Format:	Artikel
Sprache:	eng
Schlagworte:	ABE Animals atherosclerosis Cholesterol gene editing Humans hypercholesterolemia Hypercholesterolemia - genetics Hyperlipoproteinemia Type II - genetics Hyperlipoproteinemia Type II - therapy low‐density lipoprotein receptor Mice Mutation Receptors, LDL - genetics Receptors, LDL - metabolism RNA, Guide, CRISPR-Cas Systems
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