Gene transfer and genome editing for familial hypercholesterolemia

Familial hypercholesterolemia (FH) is an autosomal dominant inherited disease characterized by high circulating low-density lipoprotein (LDL) cholesterol. High circulating LDL cholesterol in FH is due to dysfunctional LDL receptors, and is mainly expressed by hepatocytes. Affected patients rapidly d...

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Bibliographic Details
Main Authors: Cesare Canepari, Alessio Cantore
Format: Article
Language:English
Published: Frontiers Media S.A. 2023-04-01
Series:Frontiers in Molecular Medicine
Subjects:
Online Access:https://www.frontiersin.org/articles/10.3389/fmmed.2023.1140997/full