Article

Targeted Epigenetic Modulation Outperforms Nuclease- and Deaminase-Based Editing for Durable <i>Pcsk9</i> Silencing in a Clinically Relevant Delivery System

2026-03-23

Abstract excerpt

Inhibition of proprotein convertase subtilisin/kexin type 9 (PCSK9) lowers low-density lipoprotein cholesterol, a major risk factor for cardiovascular disease. Although several gene therapy strategies targeting Pcsk9 have been developed, direct comparisons across modalities are limited. To address this, we systematically evaluated cytosine base editing, nuclease-based CRISPR–Cas9, and epigenetic gene editing for...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: ffadce7c-a3e0-54da-be13-cbc4eca01c6b
DOI: 10.64898/2026.03.20.713290

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Targeted Epigenetic Modulation Outperforms Nuclease- and Deaminase-Based Editing for Durable <i>Pcsk9</i> Silencing in a Clinically Relevant Delivery SystemDOI 10.64898/2026.03.20.713290Read

In Vivo Base Editing of PCSK9 (Proprotein Convertase Subtilisin/Kexin Type 9) as a Therapeutic Alternative to Genome Editing.Semantic proximity Read

In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levelsSemantic proximity Read

CRISPR-Cas12a delivery by DNA-mediated bioresponsive editing for cholesterol regulationSemantic proximity Read

A potent epigenetic editor targeting human PCSK9 for durable reduction of low-density lipoprotein cholesterol levelsSemantic proximity Read

New Approaches for Targeting PCSK9: Small-Interfering Ribonucleic Acid and Genome EditingSemantic proximity Read

Gene Editing for the Treatment of HypercholesterolemiaSemantic proximity Read

Base Editing Gene Therapy for Heterozygous Familial HypercholesterolemiaSemantic proximity Read

Moving toward genome-editing therapies for cardiovascular diseasesSemantic proximity Read

Engineered PsCas9 enables therapeutic genome editing in mouse liver with lipid nanoparticlesSemantic proximity Read

Eliminating base-editor-induced genome-wide and transcriptome-wide off-target mutations.Semantic proximity Read

A non-viral CRISPR/Cas9 delivery system for therapeutically targeting HBV DNA and pcsk9 in vivoSemantic proximity Read

Is It Ever Wise to Edit Wild-Type Alleles? Engineered CRISPR Alleles Versus Millions of Years of Human Evolution.Semantic proximity Read