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 ...

Many human diseases are caused by point mutations that affect RNA splicing. It has been observed that nonsense mutations that generate premature termination of translation products can drive the use ...

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 ...