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Updated: Apr 17, 2026
10:56
A Familial Hypercholesterolemia Human Liver Chimeric Mouse Model Using Induced Pluripotent Stem Cell-derived Hepatocytes
Published on: September 15, 2018
8.6K
[Homozygous familial hypercholesterolemia].
Summary
Familial hypercholesterolemia (FH) requires aggressive LDL-cholesterol reduction. New therapies targeting Apolipoprotein B offer hope for severe cases resistant to current treatments.
Area of Science:
- Cardiovascular Medicine
- Genetics
- Pharmacology
Context:
- Familial hypercholesterolemia (FH) involves genetic LDL receptor defects, causing severe hypercholesterolemia and early atherosclerosis.
- Standard lipid-lowering therapies, including statins, are often insufficient for homozygous FH patients.
- LDL apheresis is effective but invasive, with compliance issues and residual atherosclerosis risk.
Purpose:
- To review emerging therapeutic strategies for Familial hypercholesterolemia.
- To discuss novel treatments targeting Apolipoprotein B (ApoB).
Summary:
- This review focuses on new treatments for FH, including Lomitapide (a MTTP inhibitor) and Mipomersen (an oligonucleotide).
- These agents target ApoB, offering potential efficacy even in receptor-negative homozygous FH patients.
- They address the unmet need for effective FH treatments beyond current modalities.
Impact:
- These novel ApoB-targeted therapies may significantly improve outcomes for severe FH patients.
- They represent a promising advancement in managing genetic dyslipidemias and preventing cardiovascular events.
- Successful implementation could reduce the burden of premature atherosclerosis in FH populations.
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