Effect of adeno-associated virus-mediated transfer of low density lipoprotein receptor gene on treatment of hypercholesterolemia.
Low density lipoprotein receptor (LDL-R) plays an important role in removing LDL from plasma and preventing prbducing atherosclerosis. To test hepatic reconstitution of LDLR expression is sufficient for metabolic correction of subjects with hypercholesterolemia; a recombinant adeno-associated virus (rAAV) carrying the human LDL receptor cDNA (AAV/LDL-R) was constructed. Using adenosome, a complex of ademviral capsid protein and lipsome, to transfer the rAAV into livers of rabbits with experimental hypercholesterolemia could elicit the production of LDL-R and lower plasma cholesterol level. These results demonstrate that AAV-mediated transfer of LDL-R gene may provide an alternative means to gene therapy of hypercholesterolemia.
Chen
G
G
Institute of Cardiovascular Research, Beijing Medical University, 100083, Beijing, China.
Song
L
L
Zhu
X
X
Zhang
X
X
Zhang
C
C
Wang
T
T
Tang
J
J