The present disclosure relates to compositions and methods for inhibiting nonsense-mediated mRNA decay in a gene-specific manner, for example in the treatment of diseases or disorders caused by nonsense mutations.
本公开涉及以
基因特异性方式抑制无义介导的 mRNA 衰变的组合物和方法,例如用于治疗由无义突变引起的疾病或失调。