Background
Carnegie Mellon University, BS, 2019
Chemistry/Biomedical Eng

Current Research
Cystic Fibrosis (CF) is an autosomal recessive genetic disease that means people with it have trouble making a protein
known as CFTR. Although novel therapeutics can help the CFTR protein work better, these medicines don’t help people that make no protein at all. These people make up about 10% of all people that have the disease, and their disease is often especially bad and gets worse quicker because there are less medicines to help. These people deserve a possible way to treat their disease. We believe the best way to do this is to correct the genetic mutation itself. Although traditional gene editing technology can have many side effects, new modified technology is now more precise and less risky. Our lab has already been successful in fixing human airway cells from people with rare mutations and seeing them able to produce this protein. However, we need a method that can deliver gene editing technology safely to people. Nanoparticles have become very popular to deliver medicine and have shown to be safe as part of things like vaccines. CF is a lifelong disease that involves many organs, and while most early death is caused by lung disease, other organs such as the pancreas and intestines can lead to more health issues. Since CF affects the whole body, we think it would be best to deliver our treatment to the whole body so that other organs can possibly be helped by the treatment. We propose to determine whether nanoparticles can deliver gene editing technology to human airway cells as well as to mouse cells that naturally express the protein. Our first goal will be to determine if changes of what make up the nanoparticles allow them to be more easily taken up by cells from affected tissues in people with CF. Our second goal will be to see which organs and specific cell types take up these nanoparticles when they’re delivered to entire whole body. 

Want to learn more about nanoparticles as a therapeutic tool for gene therapy?
https://pubmed.ncbi.nlm.nih.gov/35006646/

Want to learn more about CRISPR-derived editing of rare cystic fibrosis variants?
https://pubmed.ncbi.nlm.nih.gov/37547293/

Co-PI Information
Dr. Garry Cutting
Genetic Medicine

Co-PI Information
Dr. Jordan Green
Biomedical Engineering
https://greengroup.johnshopkins.edu/