Biology
Molecular Basis of Cystic Fibrosis and CFTR Modulator Therapies
Quick fact
The most common CF mutation, ΔF508, removes a single amino acid (phenylalanine) from the CFTR protein, yet it causes the protein to fold incorrectly, be degraded, and never reach the cell surface—over 70% of people with CF carry this mutation.
Why this is interesting
You've probably heard of cystic fibrosis, but what if the real villain is a single microscopic protein that works like a faulty bouncer at a club? How can a tiny mutation cause such devastating effects?