Nonsense Suppression Therapy: An Emerging Treatment for Hereditary Skin Diseases
Keywords:readthrough, aminoglycosides, PTC124, nonsense-mediated decay, suppressor tRNA, hereditary skin diseases
Nonsense mutations cause the premature termination of protein translation via premature termination codons (PTCs), leading to the synthesis of incomplete functional proteins and causing large numbers of genetic disorders. The emergence of nonsense suppression therapy is considered to be an effective method for the treatment of hereditary diseases, but its application in hereditary skin diseases is relatively limited. This review summarizes the current research status of nonsense suppression therapy for hereditary skin diseases, and discusses the potential opportunities and challenges of applying new technologies related to nonsense suppression therapy to dermatology. Further research is needed into the possible use of nonsense suppression therapy as a strategy for the safer and specific treatment of hereditary skin diseases.
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https://doi.org/10.1002/1531-8249(200008)48:2<164::AID-ANA5>3.0.CO;2-B DOI: https://doi.org/10.1002/1531-8249(200008)48:2<164::AID-ANA5>3.0.CO;2-B
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