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Gene Therapy for Paediatric Homozygous Familial Hypercholesterolaemia.

Heart, lung & circulation - 1 Jul 2023

Graves Lara E, Horton Ari, Alexander Ian E, Srinivasan Shubha

Abstract excerpt

The clinical outcome for children and adolescents with homozygous familial hypercholesterolaemia (HoFH) can be devastating, and treatment options are limited in the presence of a null variant. In HoFH, atherosclerotic risk accumulates from birth. Gene therapy is an appealing treatment option as restoration of low-density lipoprotein receptor (LDLR) gene function could provide a cure for HoFH. A clinical trial...

Topics

Adult
Adolescent
Humans
Child
Homozygous Familial Hypercholesterolemia
Hyperlipoproteinemia Type II
Genetic Therapy
Phenotype
Mutation
Atherosclerosis

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 37012174
DOI: 10.1016/j.hlc.2023.01.017

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 31 Aug 2026.

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