CFTR mutation

Can Gene-Edited Stem Cells Treat Cystic Fibrosis?

human-IPS-cells-made-in-Knoeplfer-lab-stained-for-TRA-160

Drugs that restore the shape of the errant protein behind cystic fibrosis (CF) have, over the past eight years, helped the majority of patients, who have certain mutations. Gene-corrected stem cells might offer a “mutation agnostic” option to CF. CF results from a glitch in a glycoprotein with the unwieldy name “cystic fibrosis transmembrane conductance …

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