The Science Journal of the Lander College of Arts and Sciences
Abstract
Cystic fibrosis is a genetic disease with over 2,000 reported mutation varieties. It affects a chloride channel called CFTR, which by the transmembrane transport of chlorine, maintains an apical membrane electrolyte equilibrium in luminal epithelial cells. Its malfunction results in cystic fibrosis which causes excess mucus in various organs and is associated with respiratory complications and salty sweat. Treatment of cystic fibrosis has primarily taken two approaches: a mutation-specific method, targeted at a particular mutation to restore its function, and a mutation-non-specific method, which modulates intracellular barriers to CFTR trafficking or cellular repression of mutant protein function. This paper presents a review of the literature on the strengths and weaknesses of the most prevalent therapies for cystic fibrosis. It seeks to assess whether either approach, targeting or modulating, holds more significance and should therefore be emphasized in future research. The review concludes that despite the recent advancement and success of targeted therapies, there is good reason to continue research in both targeting and modulating therapies because they can complement each other and help increase availability while also lowering drug costs.
Recommended Citation
Yechiel Muller. (2024). Targeting vs. Modulating: How to Confront Variation in Cystic Fibrosis?. The Science Journal of the Lander College of Arts and Sciences, 17(2), 21-30. Retrieved from https://touroscholar.touro.edu/sjlcas/vol17/iss2/5
