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