In December, the FDA approved Vertex’s vanzacaftor, tezacaftor and deutivacaftor (Alyftrek) for the treatment of cystic fibrosis in patients with at least one F508del mutation or another ...

This protein machinery—the DNA mismatch repair system—reversed the prime editor’s changes if it noticed the rewriting. By adding in silent mutations near the edits they made to F508del, Liu’s team ...

There are five classes of CFTR mutations: protein production, protein processing, gating, conduction, and insufficient protein. The most common CF mutation, F508del, is primarily considered to be a ...