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news.Amsterdam Molecular Therapeutics (AMT) has said that its gene therapy product incorporating siRNA sequences into microRNA scaffolds to silence Apolipoprotein B100 (AAV-miApoB) was able to significantly lower plasma cholesterol levels in vivo over a period of 18 weeks.
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ApoB100 is the structural protein of Low Density Lipoprotein (LDL) particles that carry cholesterol. Silencing the activity of ApoB100 with miRNA or shRNA lowers plasma LDL-cholesterol by 60-80% and has the potential to be used to treat hypercholesterolemia and associated cardiovascular disease.
In the new study, mice received intra-venous injections with equal doses of 1011 gc per animal AAV-shApoB or AAV-miApoB and were examined for 18 weeks.
Expression of the shApoB and miApoB resulted in 90% ApoB protein knock-down, associated with 80% cholesterol decrease in murine plasma for the first 6 weeks.
AMT CEO Jorn Aldag said that the successful hepatocyte-specific delivery of microRNA (miRNA) and significant demonstration of gene silencing again illustrates the strength of AMT’s AAV platform.
"We announced previously success with our short hairpin RNA targeting ApoB (shApoB) gene product but our newest data suggests our miRNA product is proving to be even better and safer," Aldag said.