isfor A discussion of exon skipping presented in: Fairclough, R.J. et al. 2013 i

Question

isfor A discussion of exon skipping presented in: Fairclough, R.J. et al. 2013 ing in DMD is for use to treat familial hypercholes- terolemia. How does this ASO work Therapy for Duchenne muscular dystro-and why was its use restricted to phy: Renewed optimism from genetic approaches. Nature Rev. Genet. 14: 373zygous heredi- 378. For information about clinical trials, start your search on PubMed .ncbi.nlm.nih.gov/pubmed. 3. Mipomersen (Kynamro, Isis Pharma patients with a tary disease? heredi- To learn about mipomersen, see Bell, D.A. et al. 2012. Mipomersen and other ther- apies for the treatment of severe familial hypercholesterolemia. Vascular Health and To learn about m at http://www ceuticals) has recently been approved Risk Management 8: 651-659

Explanation / Answer

Mipomersen is a drug that decreases the level of apolipoprotein B, LDL and non-high density lipoprotein cholesterol and total cholesterol. It binds to messenger RNA coding for apolipoprotein B-100, which is a protein that is the main component of LDL and VLDL. It is restricted to patients with homozygous hereditary disease because their body is unable to remove LDL from the body. So when this drug is administered the levels of LDL can be reduced.

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