Considering these design parameters, several studies leaded by Crooke and Corey have demonstrated the effective use of ss-siRNAs to silence diverse molecular targets including metabolic regulators such as phosphatase and tensin homolog (PTEN), coagulation factor VII, and apolipoprotein CIII (ApoCIII) in hepatocytes, as well as the mutant HTT gene associated with Huntington’s disease (Figure 5b(1),c) [161,162,164,165]. This evidence concerns the gene APOC3 and juvenile Huntington disease.