A nonsense mutation is a genetic mutation in a DNA sequence that results in a shorter, unfinished protein product. DNA is a chain of many smaller molecules called nucleotides. During protein ...

Inhibition of nonsense-mediated decay turns immunologically cold tumors hot by increasing the amount of mutant RNA leading to an increase in T-cell-targetable neoantigens on the cell surface.

“[These drugs cannot] cure the root of the disease because it is a genetic disease.” The patient’s cells must produce the CFTR protein for the existing therapies to work, which leaves patients with ...