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HealthData.Gov

Taking stock of gene therapy for cystic fibrosis

The identification of the cystic fibrosis (CF) gene opened the way for gene therapy. In the ten years since then, proof of principle in vitro and then in animal models in vivo has been followed by numerous clinical…

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Updated Oct 29, 2026

HealthData.Gov

Research Article: BMC Medical Genetics

Background SFHR (small fragment homologous replacement)-mediated targeting is a process that has been used to correct specific mutations in mammalian cells. This process involves both chemical and cellular factors that…

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Updated Oct 29, 2026