Congratulations to the ISIS team for getting the first oligonucleotide drug into the clinic. It has been a long time coming, but maybe mipomersen will open the door to other molecules based on antisense, siRNA, or splice switching oligos. Kynamro (mipomersen) is an antisense that targets the RNA for apolipoprotein B100, a component of low density lipoprotein. A small cohort of people with homozygous familial hypercholesterolemia are resistant to statins and have highly elevated cholesterol levels that lead to serious cardiovascular problems at an early age. Kynamro should be able to help these individuals.
http://blogs.nature.com/news/2013/01/fda-approves-antisense-cholesterol-drug.html
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