ReviewCurrent atherosclerosis reports2016
Recent Developments in Gene Therapy for Homozygous Familial Hypercholesterolemia.
Review in Current atherosclerosis reports, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
15 citing papers in PubMed, 1 synthesis or guideline pooled it, 50 citations in OpenAlex.
- The promising novel therapies for familial hypercholesterolemia.Journal of clinical laboratory analysis · 2022Pooled it
- Efficacy and Safety of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia: Phase 3, Multinational Open-Label Study.Arteriosclerosis, thrombosis, and vascular biology · 2022Trial
- An Improved Prognosis in Subjects With Homozygous Familial Hypercholesterolemia Associated With Advances in LDL-Lowering Therapy.Journal of atherosclerosis and thrombosis · 2026Article
- Gene therapy for liver diseases: methods, challenges and opportunities.Journal of nanobiotechnology · 2026Review
- Prevalence of Carotid Atherosclerotic Plaques and Stenosis in Adults with Familial Hypercholesterolemia Needs Reappraisal: Systematic Review and Meta-Analysis.Journal of clinical medicine · 2025Review
- Established and Emerging Nucleic Acid Therapies for Familial Hypercholesterolemia.Circulation · 2024Review
- Significance of traditional herbal medicine for dyslipidemia.American journal of translational research · 2023Review
- New Trends and Therapies for Familial Hypercholesterolemia.Journal of clinical medicine · 2022Review
- Perioperative Management and Clinical Outcomes of Liver Transplantation for Children with Homozygous Familial Hypercholesterolemia.Medicina (Kaunas, Lithuania) · 2022Article
- Familial hypercholesterolaemia: evolving knowledge for designing adaptive models of care.Nature reviews. Cardiology · 2020Review
- Efficient Gene Transfer to the Central Nervous System by Single-Stranded Anc80L65.Molecular therapy. Methods & clinical development · 2018Article
- Somatic Editing of Ldlr With Adeno-Associated Viral-CRISPR Is an Efficient Tool for Atherosclerosis Research.Arteriosclerosis, thrombosis, and vascular biology · 2018Article
- Novel Approaches for the Treatment of Familial Hypercholesterolemia: Current Status and Future Challenges.Journal of atherosclerosis and thrombosis · 2018Review
- AAV9-NPC1 significantly ameliorates Purkinje cell death and behavioral abnormalities in mouse NPC disease.Journal of lipid research · 2017Article
- Gene Delivery in Lipid Research and Therapies.Methodist DeBakey cardiovascular journalReview
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 1 institution in 1 country.
Funding
Abstract
Homozygous familial hypercholesterolemia (HoFH) is a life-threatening Mendelian disorder with a mean life expectancy of 33 years despite maximally tolerated standard lipid-lowering therapies. This disease is an ideal candidate for gene therapy, and in the last few years, a number of exciting developments have brought this approach closer to the clinic than ever before. In this review, we discuss in detail the most advanced of these developments, a recombinant adeno-associated virus (AAV) vector carrying a low-density lipoprotein receptor (LDLR) transgene which has recently entered phase 1/2a testing. We also review ongoing development of approaches to enhance transgene expression, improve the efficiency of hepatocyte transduction, and minimize the AAV capsid-specific adaptive immune response. We include a summary of key gene therapy approaches for HoFH in pre-clinical development, including RNA silencing of the gene encoding HMG-CoA reductase (HMGCR) and induced pluripotent stem cell transplant therapy.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.