Maria Hudock
Cracking the Case on Gene Therapy for Cystic Fibrosis
Summary
Cystic fibrosis (CF) is a genetic disease that causes severe lung damage over time, sometimes requiring a lung transplant or resulting in early death. Researchers have been working to create a gene therapy that would correct the genetic mutation that causes CF. However, gene therapies that have worked in animals and in human lung cells in the lab have not worked in humans. Why? Research shows that endocytosis, the process by which cells take up large objects (like gene therapy carriers), works differently in animals than in humans. It also works differently in human cells outside vs. inside the body. Using a tool that I custom-built to keep small pieces of lung alive outside the body, I will compare endocytosis mechanisms in animal and human tissue to understand exactly how this process differs, so we can better predict which gene therapies will be successful in humans. I will also identify which carrier properties (size, shape, etc.) result in more cell uptake, so we can design better gene therapy carriers.
Receiving this fellowship has increased my confidence as an investigator by affirming that I can generate interesting, relevant project ideas, by helping me to practice study planning, and by bolstering me to follow through and work with even more conviction. For this, I am extremely grateful.
