2 datasets found
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Federal
Taking stock of gene therapy for cystic fibrosis
U.S. Department of Health & Human Services —
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... -
Federal
Research Article: BMC Medical Genetics
U.S. Department of Health & Human Services —
Background SFHR (small fragment homologous replacement)-mediated targeting is a process that has been used to correct specific mutations in mammalian cells. This...
2 datasets found