Target Validation Information
TTD ID	T09507
Target Name	APOB messenger RNA (APOB mRNA)
Type of Target	Successful
Action against Disease Model	Mipomersen	Drug Info	The hybridization of ISIS 301012 to apoB-100 mRNA results in RNase H-mediated degradation of the cognate mRNA, thereby inhibiting the translation of the apoB protein. ApoB-100 is the principal apolipoprotein of very low-density lipoprotein cholesterol, intermediate-density lipoprotein cholesterol, and low-density lipoprotein cholesterol.Thus, apoB-100 inhibition is a promising target for the development of new treatment options to lower low-density lipoprotein cholesterol. ISIS 301012 represents the first antisense compound of the lipid-lowering class to be dosed in h uManThe in vitro pharmacological activity of ISIS 301012 had been demonstrated in several h uMan cell lines(HepG2 and Hep3B)and primary h uMan hepatocytes, where IC50 values were in the range of 10 to 50 nM for target mRNA reduction.	[1]
References
REF 1	Cross-species pharmacokinetic comparison from mouse to man of a second-generation antisense oligonucleotide, ISIS 301012, targeting human apolipopr... Drug Metab Dispos. 2007 Mar;35(3):460-8.

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