Idera announced new data on its novel gene-silencing oligonucleotide
(GSO) technology at the Cell Symposium on Regulatory RNAs in Chicago,
IL. In preclinical studies, systemic delivery of GSOs targeted to ApoB
or PCSK9 mRNA caused a reduction in the level of the targeted mRNA and
associated protein and resulted in a decrease in serum total cholesterol and LDL-cholesterol concentration. ApoB and PCSK9 are two validated targets associated with cardiovascular diseases.
In this study, Idera created 19mer GSOs for apolipoprotein B (ApoB) and proprotein convertase subtilisin/kexin type 9 (PCSK9) mRNA and evaluated their in vivo activity in mice following subcutaneous administration. The data demonstrate that treatment with each GSO led to a significant reduction in the concentration of the target associated mRNAs and protein. The effects were specific, with no significant effects being observed on ABCA1, ABCG1 or LXR mRNA levels. In addition, treatment with GSOs for either ApoB or PCSK9 resulted in a decrease in total serum cholesterol and LDL-cholesterol.
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In this study, Idera created 19mer GSOs for apolipoprotein B (ApoB) and proprotein convertase subtilisin/kexin type 9 (PCSK9) mRNA and evaluated their in vivo activity in mice following subcutaneous administration. The data demonstrate that treatment with each GSO led to a significant reduction in the concentration of the target associated mRNAs and protein. The effects were specific, with no significant effects being observed on ABCA1, ABCG1 or LXR mRNA levels. In addition, treatment with GSOs for either ApoB or PCSK9 resulted in a decrease in total serum cholesterol and LDL-cholesterol.
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