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A large number of genetic diseases, such as cystic fibrosis, come about from mutations that cause nonsense sequences to form, often with a termination sequence being created too early in the protein's code.
Rochester University scientists have developed specialized guide RNAs to fix these terminations and also suppress the nonsense-mediated mRNA decay system that turns off the protein coding.
#Medicine #Disease #CysticFibrosis #Genetics #Biotech #mRNA #Science #Scicomm
https://www.cell.com/molecular-cell/fulltext/S1097-2765(23)00029-1#%20
Molecular CellTargeted pseudouridylation: An approach for suppressing nonsense mutations in disease genesAdachi et al. have developed a new approach (targeted PTC pseudouridylation) to nonsense
suppression. Targeted PTC pseudouridylation is achieved by introducing into cells
a designer box H/ACA RNA targeting the mRNA PTC. The conversion of the uridine to
pseudouridine at the PTC suppresses NMD and promotes PTC readthrough, restoring full-length
protein.