Promising Therapies for Cystic Fibrosis

This section discusses the potential of therapies to treat the genetic cause of cystic fibrosis by restoring CFTR function in the lungs.

How Therapeutics Work

• Therapeutics can be delivered directly to cells through the airway.

• Highly specialized cells that line the respiratory tract help protect airways from invading microbes, infection, and injury.

• The CFTR protein helps control the movement of salt and fluids into and out of the cell to keep mucus at a healthy level.

• Reprogramming cells to make normal CFTR protein is possible by providing correct instructions.

Potential Treatments

• RNA therapy and gene therapy are two potential treatments that can deliver a correct copy of genetic instructions to cells in the lungs.

• Regular repeated treatments will be needed as cells naturally die and are replaced.

• Gene editing is a potential therapy that would permanently repair the CFDR gene mutation in cellular DNA.

• Cell-based therapy involves removing cells from the airway to create space for delivery of gene-edited cells.

Scientific Progress

• Scientific progress in genetic-based therapies is advancing at an unprecedented pace.

• It will take time and investment to develop these treatments that could lead to restoring CFTR function not just in the lung but also in all parts of the body affected by cystic fibrosis.

Conclusion

This section concludes with a commitment from The Foundation to ensure everyone with cystic fibrosis has a treatment for their underlying disease cause, and one day a cure.

• The Foundation is committed to ensuring that everyone with cystic fibrosis has a treatment for the underlying cause of their disease and one day a cure.