BIO 115 Lecture Notes - Lecture 25: Lumacaftor, Wild Type, Safety Data Sheet
Document Summary
Differentiate between novel cf treatments based on mutations they are targeting. Gentamicin, an aminoglycoside antibiotic for class 1 mutations. A large phase 3 trial testing an aminoglycoside analog, ptc 124, was conducted in patients with nonsense mutations and showed modest success. Vx-770 or ivacaftor (kalydeco) for class 3 mutations. Augments chloride channel function for mutant and wild-type cftr that sits in the plasma membrane. Shown to improve lung function and nutritional status as well as to significantly reduce sweat cl- concentration in patients who have the. G551d (class 3) mutations and other class 3 mutations and the r117 mutations. May be effective for class 4 mutations. lumacaftor + ivacaftor for f508 (class 2) mutations. The f508del mutation causes the mutant misfolded cftr protein to be. Trapped in the endoplasmic reticulum, and then degraded in the cell. In addition f508del cftr also displays abnormal gating functions. Evaluation of a patient with possible occupational lung disease.
