- Tytuł:
-
Emerging Personalized Opportunities for Enhancing
Translational Readthrough in Rare Genetic Diseases and Beyond. - Autorzy:
- Źródło:
- International journal of molecular sciences [Int J Mol Sci] 2023 Mar 23; Vol. 24 (7). Date of Electronic Publication: 2023 Mar 23.
- Typ publikacji:
- Journal Article; Review
- MeSH Terms:
-
Codon, Nonsense*/genetics
Genetic Diseases, Inborn*/genetics
Genetic Diseases, Inborn*/therapyPeptide Chain Elongation ,Translational */drug effects
Precision Medicine*/methods
Precision Medicine*/trends
Rare Diseases*/genetics
Rare Diseases*/therapy
Suppression, Genetic*/drug effects
Suppression, Genetic*/genetics
Animals ; Humans ; Breast Neoplasms/genetics ; Breast Neoplasms/therapy ; Cystic Fibrosis/genetics ; Cystic Fibrosis/therapy ; Epidermolysis Bullosa/genetics ; Epidermolysis Bullosa/therapy ; Nephritis, Hereditary/genetics ; Nephritis, Hereditary/therapy ; Nonsense Mediated mRNA Decay ; Retinitis Pigmentosa/genetics ; Retinitis Pigmentosa/therapy ; Shwachman-Diamond Syndrome/genetics ; Shwachman-Diamond Syndrome/therapy ;Peptide Chain Termination,Translational /drug effects ; Aminoglycosides/pharmacology
Czasopismo naukowe