Hypercholesterolemia in low density lipoprotein receptor knockout mice and its reversal by adenovirus-mediated gene delivery.
Authors
Discussion
Loading...
Member takes
Implication
Randomized trial reveals gene therapy reduces cholesterol levels in hypercholesterolemic mice, indicating potential for treatment.
Key Points
- This research aims to investigate hypercholesterolemia in LDL receptor knockout mice and explore gene therapy as a reversal method.
- Created LDL receptor knockout mice using homologous recombination in embryonic stem cells.
- Administered a recombinant adenovirus encoding the human LDL receptor to evaluate cholesterol level reversal.
- Measured plasma cholesterol levels, particle size, and receptor activity after gene delivery.
- LDLR-/- mice exhibited total plasma cholesterol levels twofold higher than wild-type mice.
- Adenovirus delivery normalized elevated IDL and LDL levels within 4 days, restoring LDL receptor function.
- Increased clearance of 125I-VLDL in treated LDLR-/- mice confirmed gene therapy efficacy.
Cite This Study
Ishibashi et al. (1993) studied this question.