"A single multipurpose gene-editing tool can correct several genetic conditions by restoring proteins that have been truncated by disease-causing mutations. The method might one day overcome a key stumbling block faced by gene-editing therapies: the need to design a bespoke treatment for each disease. The new approach, called PERT, combines gene editing with engineered RNA molecules that allow protein synthesis to continue even when a mutation in the DNA tells it to stop prematurely."
"So far, PERT has overcome disease-linked nonsense mutations in mice and in human cells grown in culture, but further testing and refinement are needed before the approach can be studied in people, says David Liu, a chemical biologist at the Broad Institute in Cambridge, Massachusetts, and a co-author of a paper about the technique. If it does prove effective in humans, PERT could drive down the cost and speed up the development of gene-editing therapies for many conditions."
PERT combines prime editing with engineered suppressor tRNAs to permit ribosomes to read through premature stop codons and produce full-length proteins. Nonsense mutations create premature stop signals and account for nearly one-quarter of known disease-causing DNA variants. PERT restored proteins in mice and in human cells grown in culture, correcting disease-linked nonsense mutations. Additional testing and refinement are required before the approach can be evaluated in humans. If effective in people, PERT could lower costs and speed development of gene-editing therapies by providing a disease-agnostic strategy that avoids bespoke treatments for each condition.
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