Prime Editing–mediated Readthrough: Single Genome-Editing Strategy for Genetic Disorders

Context
Scientists have found a single genome-editing method that can correct many genetic disorders caused by nonsense mutations (a type of DNA error that stops protein production too early).

Key Idea: One Strategy for Many Disorders
Researchers from Harvard, Broad Institute, and University of Minnesota developed a method called Prime Editing–mediated Readthrough (PERT).
It reprograms the cell’s own machinery so it can ignore the faulty stop signal and make the full, correct protein.

What is tRNA?
• tRNA (transfer RNA) is a small molecule that reads mRNA code and brings the correct amino acids to build proteins.
• It acts like a translator during protein synthesis.
• Scientists engineered a special suppressor tRNA that can override premature stop codons, allowing cells to finish making the protein.

Why it matters
• Nonsense mutations cause ~25% of all genetic disorders (e.g., cystic fibrosis, Tay-Sachs).
• Each mutation usually needs a different therapy — slow and expensive.
• This single strategy could treat multiple diseases with the same toolkit.

PYQ – 2019, Ans – A
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Prime Editing–mediated Readthrough: Single Genome-Editing Strategy for Genetic Disorders

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