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2. New approaches to the treatment of orphan genetic disorders: Mitigating molecular pathologies using chemicals
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3. A Protein Biosynthesis Machinery Strategy for Identifying P53PTC -Rescuing Compounds as Synergic Anti-Tumor Drugs
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4. Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses of ELX-02, a Potential Treatment for Genetic Disorders Caused by Nonsense Mutations, in Healthy Volunteers
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5. Therapeutic developments for Duchenne muscular dystrophy
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6. Strategies against Nonsense: Oxadiazoles as Translational Readthrough-Inducing Drugs (TRIDs)
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7. Enhancement of premature stop codon readthrough in the CFTR gene by Ataluren (PTC124) derivatives
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8. Gene-targeting pharmaceuticals for single-gene disorders
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9. Mendelian Disorders of Cornification Caused by Defects in Intracellular Calcium Pumps: Mutation Update and Database for Variants in ATP2A2 and ATP2C1 Associated with Darier Disease and Hailey-Hailey Disease
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10. Impact of RNA Modifications and RNA-Modifying Enzymes on Eukaryotic Ribonucleases
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11. A Direct Approach to Decoration of Bioactive Compounds via C–H Amination Reaction
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12. Frontotemporal dementia non-sense mutation of progranulin rescued by aminoglycosides
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13. Suppression of Nonsense Mutations by New Emerging Technologies
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14. Pseudouridine-mediated stop codon readthrough in S. cerevisiae is sequence context–independent
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15. MMADHC premature termination codons in the pathogenesis of cobalamin D disorder: Potential of translational readthrough reconstitution.
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16. Therapeutic promise of engineered nonsense suppressor tRNAs
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17. Restoration of dystrophin expression in mice by suppressing a nonsense mutation through the incorporation of unnatural amino acids
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18. Ataluren—Promising Therapeutic Premature Termination Codon Readthrough Frontrunner
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19. A systematic dissection of determinants and consequences of snoRNA-guided pseudouridylation of human mRNA
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20. Premature termination codon: a tunable protein translation approach
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21. Resolving Ultrafast Photoinitiated Dynamics of the Hachimoji 5‐aza‐7‐deazaguanine Nucleobase: Impact of Synthetically Expanding the Genetic Alphabet 1
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22. Nonsense codons suppression. An acute toxicity study of three optimized TRIDs in murine model, safety and tolerability evaluation
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23. Ovarian cancer onset across differentBRCAmutation types: a view to a more tailored approach forBRCAmutated patients
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24. Mini-dCas13X–mediated RNA editing restores dystrophin expression in a humanized mouse model of Duchenne muscular dystrophy
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25. Targeted pseudouridylation: An approach for suppressing nonsense mutations in disease genes
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26. Targeted Pseudouridylation: A Novel Approach for Suppressing Nonsense Mutations in Disease Genes
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27. Advanced genetic therapies for the treatment of Rett syndrome: state of the art and future perspectives
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28. Noncanonical Amino Acid Incorporation in Mice
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29. Applications of genetic code expansion technology in eukaryotes
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30. Special issue on nucleic acid photophysics
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