From a young age, I was determined that cystic fibrosis would never stop me from pursuing my dreams in the medical field. It wasn’t until I hit a road bump in pharmacy school that I realized my CF could actually give me a unique advantage.
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As cystic fibrosis damaged my lungs, I did all I could to make it to my university graduation before I underwent lung transplant.
mRNA therapy is one way to deliver the correct genetic instructions to cells, which would allow them to make functional CFTR protein regardless of an individual’s CF mutations.
Cystic fibrosis is caused by mutations in the gene responsible for producing the cystic fibrosis transmembrane conductance regulator (CFTR) protein. For this reason, scientists are exploring ways to provide a correct copy of the gene to treat CF.
Cystic fibrosis is caused by mutations in both copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Scientists are examining whether it is possible to correct the mutations through a process called gene editing.