Nucleic Acids as Therapeutic Agents - icaam

Fig. 11.20 A conjugate of cholesterol and a siRNA where the cholesterol iscoupled through the 5’-OH of the sense strand of the siRNA. Followinginjection of the complex into mice, cholesterol facilitates uptake of thesiRNA into specific tissues and silencing of apolipoprotein B (which isinvolved in cholesterol metabolism). The antisense strand becomes part ofthe RISC complex and specifies where the mRNA is to be cleaved. In mice,target mRNA was decreased significantly in the liver and the jejunum (partof the small intestine) thereby reducing serum cholesterol levels.