Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Nonclinical Pharmacology/Toxicology Study of AAV8.TBG.mLDLR and AAV8.TBG.hLDLR in a Mouse Model of Homozygous Familial Hypercholesterolemia

1 Mar 2017

Abstract excerpt

The homozygous form of familial hypercholesterolemia (HoFH) is an excellent model for developing in vivo gene therapy in humans. The success of orthotropic liver transplantation in correcting the metabolic abnormalities in HoFH suggests that the correction of low-density lipoprotein receptor (LDLR) expression in hepatocytes via gene therapy should be sufficient for therapeutic efficacy. Vectors based on...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to CRISPR and Genetic Engineering
Share to Viral Infections and Immunology Research
Share to Virus-based gene therapy research

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 28319445
DOI: 10.1089/humc.2017.007

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 2 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Nonclinical Pharmacology/Toxicology Study of AAV8.TBG.mLDLR and AAV8.TBG.hLDLR in a Mouse Model of Homozygous Familial HypercholesterolemiaPMID 28319445Read ↗

Biodistribution of AAV8 Vectors Expressing Human Low-Density Lipoprotein Receptor in a Mouse Model of Homozygous Familial HypercholesterolemiaSemantic proximity Read ↗

Adeno-Associated Virus Serotype 8 Gene Therapy Leads to Significant Lowering of Plasma Cholesterol Levels in Humanized Mouse Models of Homozygous and Heterozygous Familial HypercholesterolemiaSemantic proximity Read ↗

Non-Clinical Study Examining AAV8.TBG.hLDLR Vector-Associated Toxicity in Chow-Fed Wild-Type and LDLR+/- Rhesus Macaques.Semantic proximity Read ↗

Gene Therapy for Familial HypercholesterolemiaSemantic proximity Read ↗

Gene Therapy in a Humanized Mouse Model of Familial Hypercholesterolemia Leads to Marked Regression of AtherosclerosisSemantic proximity Read ↗

Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemiaSemantic proximity Read ↗

[The safety and efficacy of adeno-associated virus-mediated LDLR transfection in homozygous familial hypercholesterolemia].Semantic proximity Read ↗

Safety and Feasibility of Liver-Directed Ex Vivo Gene Therapy for Homozygous Familial HypercholesterolemiaSemantic proximity Read ↗

Gene therapy with novel adeno‐associated virus vectors substantially diminishes atherosclerosis in a murine model of familial hypercholesterolemiaSemantic proximity Read ↗

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

In vivo adeno-associated virus-mediated LDLR/PCSK9 intervention for familial hypercholesterolemiaSemantic proximity Read ↗

Sleeping Beauty Transposon Vectors in Liver-directed Gene Delivery of LDLR and VLDLR for Gene Therapy of Familial HypercholesterolemiaSemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.