Nohemy Celis
Advisor: Martin Burke
Cohort: 2022

Numerous diseases arise from a missing or dysfunctional protein, but what if a small molecule was used as a replacement for the critical protein? The Burke laboratory took a unique approach to this issue and utilized the channel forming properties of the naturally occurring anti-fungal Amphotericin B (AmB), to use as a molecular prosthetic and study its influence on the genetic disease, Cystic Fibrosis. Cystic Fibrosis is caused by a missing or dysfunctional anion channel responsible for the secretion of Cl and regulation of HCO3. Currently, there are modulators that address 90% of mutations responsible for the disease. However, the benefit from these modulators, especially for those with the more severe mutation, is limited. My research interest is twofold: first, I want to understand how the molecular prosthetic, AmB, compares to the current modulators and better understand its potential to help the 10% of people that do not benefit from modulators. Second, it is to investigate whether there are additive effects when AmB is combined with the current modulators to enhance the therapeutic effect of the 90% of people with Cystic Fibrosis.