The Mutation Analysis Program provides free genetic testing to people with cystic fibrosis to help identify their CF gene variants.
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As people with cystic fibrosis live longer, there is a growing demand for physicians who are trained to address the unique medical needs of adults with CF and a need for greater participation from adult care programs in the clinical research enterprise.
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.