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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.
Clinical trials are critical to developing new treatments for CF. So, how exactly does an experimental drug become an approved therapy?
There have never been more opportunities to help develop new drugs for cystic fibrosis than there are today. When you are deciding whether to participate in a
When participating in a clinical trial, nothing is more important than your safety. We go the extra mile when it comes to protecting study volunteers.
Learn how your child's