Related Experiment Video

Updated: May 29, 2026

A Familial Hypercholesterolemia Human Liver Chimeric Mouse Model Using Induced Pluripotent Stem Cell-derived Hepatocytes
10:56

A Familial Hypercholesterolemia Human Liver Chimeric Mouse Model Using Induced Pluripotent Stem Cell-derived Hepatocytes

Published on: September 15, 2018

Familial hypercholesterolemia: present and future management

B Sjouke1, D M Kusters, J J P Kastelein

  • 1Department of Vascular Medicine, Academic Medical Center, Amsterdam, The Netherlands.

Current Cardiology Reports
|September 23, 2011
PubMed

Insights

Familial hypercholesterolemia (FH) patients need new treatments beyond statins to lower low-density lipoprotein cholesterol (LDL-C) and prevent cardiovascular disease (CVD). Emerging therapies, including mRNA antisense, show promise for managing severe dyslipidemia in FH.

Area of Science:

  • Cardiovascular Medicine
  • Metabolic Disorders
  • Pharmacology

Background:

  • Familial hypercholesterolemia (FH) is a genetic disorder causing high LDL-C levels.
  • Elevated LDL-C significantly increases the risk of premature cardiovascular disease (CVD).
  • Current treatments, primarily statins, may not be sufficient for all FH patients.

Purpose of the Study:

  • To review advancements in understanding LDL metabolism.
  • To explore novel therapeutic targets and treatment modalities for FH.
  • To assess the potential of emerging therapies in managing FH.

Main Methods:

  • Review of recent scientific literature on FH, LDL metabolism, and novel therapies.
  • Analysis of emerging treatment modalities, including mRNA antisense therapy.
  • Evaluation of clinical evidence for novel agents in lowering LDL-C.

Main Results:

  • Significant progress has been made in identifying new therapeutic targets for FH.
  • Novel treatment modalities, such as mRNA antisense therapy, are emerging.
  • Some new therapies demonstrate effectiveness in reducing plasma LDL-C levels.

Conclusions:

  • Emerging therapies hold promise for FH patients, particularly those unresponsive to or intolerant of statins.
  • New treatments are expected to enhance the management of severe dyslipidemia in FH.
  • Advancements in LDL metabolism research are paving the way for future therapeutic strategies against CVD.

More Related Videos

LDL Cholesterol Uptake Assay Using Live Cell Imaging Analysis with Cell Health Monitoring08:45

LDL Cholesterol Uptake Assay Using Live Cell Imaging Analysis with Cell Health Monitoring

Published on: November 17, 2018

Related Concept Videos

Atherosclerosis III: Management01:26

Atherosclerosis III: Management

Management of atherosclerosis involves an integrated strategy encompassing pharmacological treatment, surgical interventions, lifestyle changes, and nutrition therapy to address the multifactorial nature of the disease.Pharmacological TherapyA cornerstone of atherosclerosis management is the use of pharmacological agents. Statins, such as atorvastatin, are pivotal in inhibiting HMG-CoA reductase, an enzyme that catalyzes an initial step in cholesterol synthesis in the liver. This reduction in...
Cholesterol: Significance and Regulation01:29

Cholesterol: Significance and Regulation

Although not a source of energy, cholesterol plays a significant role as a foundational structure for bile salts, steroid hormones, and vitamin D, as well as being a crucial component of plasma membranes. Approximately 15% of blood cholesterol is derived from our diet, with the remainder synthesized from acetyl CoA by the liver and intestines. Cholesterol is eliminated from the body through its conversion into bile salts, which are eventually discarded in the feces.
Considering cholesterol and...
Pharmacogenomics: Identification of New Drug Targets01:29

Pharmacogenomics: Identification of New Drug Targets

Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
Atherosclerosis IV: Nursing Management01:23

Atherosclerosis IV: Nursing Management

Nursing management for a patient with arteriosclerosis involves a comprehensive approach focusing on lifestyle modification, disease monitoring, education, and symptomatic care. Here is an overview of effective nursing strategies:Assessment and Monitoring: Initial and ongoing assessments are crucial. Nurses must document the patient's medical history, including any hypertension, diabetes, hyperlipidemia, and other cardiovascular diseases. Assessments also cover family history and lifestyle...
Lipid-Lowering Drugs: Statins and Miscellaneous Agents01:20

Lipid-Lowering Drugs: Statins and Miscellaneous Agents

Hyperlipidemia, a medical condition often referred to as high cholesterol, is characterized by abnormally elevated levels of lipids in the bloodstream. When present in excess, these lipids, specifically cholesterol and triglycerides, can lead to serious health complications, often involving cardiovascular diseases. Illnesses like atherosclerosis, heart attacks, and pancreatitis have all been linked to untreated hyperlipidemia. This means controlling and regulating cholesterol and triglyceride...
Cystic Fibrosis: Management01:24

Cystic Fibrosis: Management

Cystic fibrosis (CF) is an autosomal recessive disorder that predominantly affects individuals of Northern European descent, occurring at a rate of 1 in 3500. It is caused by a genetic mutation in a gene on chromosome 7, most commonly the ΔF508 mutation, that codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This results in thicker mucus secretions and obstruction pathologies in multiple organs, including the lungs and sinuses.
Sinus disease and chronic sinusitis...