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=== Prime editing-mediated readthrough of premature termination codons (PERT) === |
=== Prime editing-mediated readthrough of premature termination codons (PERT) === |
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Prime editing-mediated readthrough of premature termination codons (PERT) is a genome-editing strategy to correct nonsense mutations. The method converts non-essential endogenous transfer RNA (tRNA) into a suppressor tRNA. Upon genome translation this allows readthrough of premature stop codons. The mechanism targets the general molecular mechanism of ribosomal recognition of premature stop codons and is therefore described to be applicable independent of the disease. <ref>{{cite journal | last=Pierce | title=Prime editing-installed suppressor tRNAs for disease-agnostic genome editing | journal=Nature | doi=10.1038/s41586-025-09732-2 | url = https://www.nature.com/articles/s41586-025-09732-2 | access-date=13 December 2025}}</ref> Prime editing is one of the three classes of genome-editing technologies in mammalian cells. The other two are CRISPR-associated nucleases and base editors. <ref>{{cite journal | last=Zhao |title=Prime editing: advances and therapeutic applications | url=https://www.cell.com/trends/biotechnology/fulltext/S0167-7799(23)00085-9?_returnURL=https%3A%2F%2Flinkinghub.elsevier.com%2Fretrieve%2Fpii%2FS0167779923000859%3Fshowall%3Dtrue | doi=10.1016/j.tibtech.2023.03.004 | journal = Trends in Biotechnology | access-date=13 December 2025}}</ref> |
Prime editing-mediated readthrough of premature termination codons (PERT) is a genome-editing strategy to correct nonsense mutations. The method converts non-essential endogenous transfer RNA (tRNA) into a suppressor tRNA. Upon genome translation this allows readthrough of premature stop codons. The mechanism targets the general molecular mechanism of ribosomal recognition of premature stop codons and is therefore described to be applicable independent of the disease. <ref>{{cite journal | last=Pierce | title=Prime editing-installed suppressor tRNAs for disease-agnostic genome editing | journal=Nature | doi=10.1038/s41586-025-09732-2 | url = https://www.nature.com/articles/s41586-025-09732-2 | access-date=13 December 2025}}</ref> Prime editing is one of the three classes of genome-editing technologies in mammalian cells. The other two are CRISPR-associated nucleases and base editors. <ref>{{cite journal | last=Zhao |title=Prime editing: advances and therapeutic applications | url=https://www.cell.com/trends/biotechnology/fulltext/S0167-7799(23)00085-9?_returnURL=https%3A%2F%2Flinkinghub.elsevier.com%2Fretrieve%2Fpii%2FS0167779923000859%3Fshowall%3Dtrue | doi=10.1016/j.tibtech.2023.03.004 | journal = Trends in Biotechnology | access-date=13 December 2025}}</ref> |
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=== Principle of the method === |
=== Principle of the method === |
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Many genetic diseases, such as Cystic fibrosis and Duchenne muscular dystrophy are caused by nonsense mutations in a gene. This causes the production of a non-functional protein via protein translation. PERT uses a method called prime editing. Small changes inside living cells can be made by programmable nickase fused to a polymerase and a small guide RNA. <ref>{{cite journal | last=Chen | title=Prime editing for precise and highly versatile genome manipulation |url=https://pmc.ncbi.nlm.nih.gov/articles/PMC10989687/ | journal= Nat Rev Genet| doi=10.1038/s41576-022-00541-1 |access-date=13 December 2025}}</ref> |
Many genetic diseases, such as Cystic fibrosis and Duchenne muscular dystrophy are caused by nonsense mutations in a gene. This causes the production of a non-functional protein via protein translation. PERT uses a method called prime editing. Small changes inside living cells can be made by programmable nickase fused to a polymerase and a small guide RNA. <ref>{{cite journal | last=Chen | title=Prime editing for precise and highly versatile genome manipulation |url=https://pmc.ncbi.nlm.nih.gov/articles/PMC10989687/ | journal= Nat Rev Genet| doi=10.1038/s41576-022-00541-1 |access-date=13 December 2025}}</ref> |
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Latest revision as of 18:59, 13 December 2025
Prime editing-mediated readthrough of premature termination codons (PERT)
[edit]
Prime editing-mediated readthrough of premature termination codons (PERT) is a genome-editing strategy to correct nonsense mutations. The method converts non-essential endogenous transfer RNA (tRNA) into a suppressor tRNA. Upon genome translation this allows readthrough of premature stop codons. The mechanism targets the general molecular mechanism of ribosomal recognition of premature stop codons and is therefore described to be applicable independent of the disease. [1] Prime editing is one of the three classes of genome-editing technologies in mammalian cells. The other two are CRISPR-associated nucleases and base editors. [2]
Principle of the method
[edit]
Many genetic diseases, such as Cystic fibrosis and Duchenne muscular dystrophy are caused by nonsense mutations in a gene. This causes the production of a non-functional protein via protein translation. PERT uses a method called prime editing. Small changes inside living cells can be made by programmable nickase fused to a polymerase and a small guide RNA. [3]
