| 摘要 |
<p>The present invention relates to methods and compositions for delivering functional LDL receptor genes to liver cells. This disclosure particularly demonstrates the successful use of adenovirus-mediated gene transfer to correct defects in the low density lipoprotein (LDL) receptor gene. Intravenous injection of recombinant virus lowered plasma cholesterol levels and increased the rate of 125I-LDL clearance from the circulation in both normal mice and knockout mice lacking functional LDL receptor genes. These results demonstrate that adenovirus-mediated transfer of the LDL receptor gene provides an efficient way of augmenting LDL receptor gene function in the liver, and demonstrate a novel approach for treating familial hypercholesterolemia, a frequent dominant human genetic disorder.</p> |
