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Article

AAV vectors expressing LDLR gain-of-function variants demonstrate increased efficacy in mouse models of familial hypercholesterolemia.

Circulation research - 29 Aug 2014

Somanathan Suryanarayan, Jacobs Frank, Wang Qiang, Hanlon Alexandra L, Wilson James M, Rader Daniel J

Abstract excerpt

RATIONALE: Familial hypercholesterolemia is a genetic disorder that arises because of loss-of-function mutations in the low-density lipoprotein receptor (LDLR) and homozygous familial hypercholesterolemia is a candidate for gene therapy using adeno-associated viral vectors. Proprotein convertase subtilisin/kexin type 9 (PCSK9) and inducible degrader of LDLR (IDOL) negatively regulate LDLR protein and could dampen...

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Source

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Identifiers

PMID: 25023731
DOI: 10.1161/CIRCRESAHA.115.304008
PMCID: PMC4165546

Genes

LDLR
PCSK9

Variants

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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