Substitutive therapy has significantly ameliorated the quality of life of patients with
coagulation factor deficiencies. However, there are some limitations that support research
towards alternative therapeutic approaches. Here we focus on the rescue of coagulation
factor biosynthesis by targeting the RNA processing and translation, which would permit
restoration of the altered gene expression while maintaining the gene regulation in the
physiological tissues. The essential prerequisite of the three reported RNA‐based correction …