Restore CFTR Function

The symptoms of cystic fibrosis are caused by a defective protein, known as the cystic fibrosis transmembrane conductance regulator (CFTR). Researchers are investigating potential therapies to restore proper function to the CFTR protein or correct its production process so that a normal protein is made.

What You Need To Know

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Basics of the CFTR Protein

Cystic fibrosis occurs when the cystic fibrosis transmembrane conductance regulator (CFTR) protein is either not made correctly, or not made at all. By understanding how the protein is made, scientists have been able to develop treatments that target the protein and restore its function.

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  1. Article

    CFTR Modulator Types

    Drugs that target the underlying defect in the cystic fibrosis transmembrane conductance regulator (CFTR) protein are called CFTR modulators. The three main types of modulators are potentiators, correctors, and amplifiers.

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  2. Article

    Gene Therapy for Cystic Fibrosis

    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.

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  3. Article

    Gene Editing for Cystic Fibrosis

    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.

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  1. Article

    Gene Delivery for Cystic Fibrosis Therapy

    For gene editing and gene replacement therapies to work in cystic fibrosis, specifically engineered DNA or RNA molecules need to get inside the cells of the lung or other organs affected by CF. The process of getting these molecules into cells is referred to as gene delivery.

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  2. Article

    Stem Cells for Cystic Fibrosis Therapy

    Stem cells are a special type of cell that can divide and produce many other cell types. Scientists are studying how adult stem cells could be used to treat cystic fibrosis.

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Find a Clinical Trial

Help us blaze a trail to better treatments and a cure for CF. Find a clinical trial that may be right for you.

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