This new approach targets disorders including cystic fibrosis, muscular dystrophy, and some cancers. In lab-grown tissue from a cystic fibrosis patient who did not respond to existing drugs, researchers restored production of a full-length protein and gave Trikafta something it could act on. The patient’s cells responded when the drug was combined with engineered transfer [...] A New RNA Strategy Could Target Thousands of Genetic Disorders
This new approach targets disorders including cystic fibrosis, muscular dystrophy, and some cancers. In lab-grown tissue from a cystic fibrosis patient who did not respond to existing drugs, researchers restored production of a full-length protein and gave Trikafta something it could act on. The patient’s cells responded when the drug was combined with engineered transfer [...] 

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