FIGURE 1.
Emerging therapeutic strategies for diseases caused by frameshift and/or nonsense mutations. The main types (top panel) and features (bottom panel) of promising therapeutic modalities either already developed or in clinical and preclinical development for a range of diseases associated with PTC-causing mutations are shown. These include methods for gene replacement, RNA replacement or editing, including splicing modulators, and nonsense-suppression strategies to overcome the deleterious effects of PTC-causing mutations. Strategies to induce targeted mRNA decay in the case of dominantly inherited diseases are also shown.
