Inhalable tRNA Therapy Reverses CF Defect, Paving Way for Other Genetic Diseases
TL;DR
University of Toronto researchers engineered an inhalable, modified tRNA that can read through premature stop codons in CFTR mRNA, restoring CFTR protein in mice and persisting in lung tissue for up to 40 days; in patient-derived organoids, tRNA plus the CF drug Trikafta restored function where the drug alone failed. The study suggests tRNA therapies may complement mRNA approaches and could be extended to Duchenne muscular dystrophy and Rett syndrome, with plans to move toward clinical trials in 2–3 years.
Topics:healthscience#cystic-fibrosis#gene-therapy#inhalable-rna#nonsense-mutations#science#trna-therapy
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