#nonsense-mutations

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fromNature
1 day ago

How to fix genetic 'nonsense': versatile gene-editing tool could tackle a host of diseases

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.
Science
Medicine
fromNature
1 day ago

Prime editing-installed suppressor tRNAs for disease-agnostic genome editing - Nature

Prime editing's versatility offers scalable strategies to treat large patient groups, addressing limitations of individualized allele-specific genome-editing for thousands of pathogenic mutations.
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