Learn about cystic fibrosis, a genetic disorder that affects the lungs, pancreas, and other organs, and how to treat and live with this chronic disease.
CF is a rare genetic disease found in about 30,000 people in the U.S. If you have CF or are considering testing for it, knowing about the role of genetics in CF can help you make informed decisions about your health care.
If you or your child has just been diagnosed with cystic fibrosis, or your doctor has recommended testing for CF, you may have many questions.
Diagnosing CF is a multistep process. A complete diagnostic evaluation should include a newborn screening, a sweat chloride test, a genetic or carrier test, and a clinical evaluation at a CF Foundation-accredited care center.
Raising a child with cystic fibrosis can bring up many questions because CF affects many aspects of your child’s life. Here you’ll find resources to help you manage your child’s daily needs and find the best possible CF care.
Living with cystic fibrosis comes with many challenges, including medical, social, and financial. By learning more about how you can manage your disease every day, you can ultimately help find a balance between your busy lifestyle and your CF care.
People with CF are living longer, healthier lives than ever before. As an adult with CF, you may reach key milestones you might not have considered. Planning for these life events requires careful thought as you make decisions that may impact your life.
People with cystic fibrosis are living longer and more fulfilling lives, thanks in part to specialized CF care and a range of treatment options.
Cystic Fibrosis Foundation-accredited care centers provide expert care and specialized disease management to people living with cystic fibrosis.
We provide funding for and accredit more than 120 care centers and 53 affiliate programs nationwide. The high quality of specialized care available throughout the care center network has led to the improved length and quality of life for people with CF.
The Cystic Fibrosis Foundation provides standard care guidelines based on the latest research, medical evidence, and consultation with experts on best practices.
As a clinician, you’re critical in helping people with CF maintain their quality of life. We’re committed to helping you partner with patients and their families by providing resources you can use to improve and continue to provide high-quality care.
As part of the Cystic Fibrosis Foundation's mission to help improve the lives of people living with cystic fibrosis, the PSDC initiative taps the CF community to inform key efforts to support the management of daily care.
Your cystic fibrosis care team includes a group of CF health care professionals who partner with you to provide specialized, comprehensive CF care.
Many cystic fibrosis patients and families face complicated issues related to getting the care they need. But CF Foundation Compass makes sure that no one has to do it alone.
For many people with cystic fibrosis, dealing with insurance is as much a part of living with the disease as nebulizers and vests. Many people with CF and their families face issues related to getting the care they need, but no one has to do it alone.
The Cystic Fibrosis Foundation is the world’s leader in the search for a cure for CF and supports a broad range of research initiatives to tackle the disease from all angles.
The CF Foundation offers a number of resources for learning about clinical trials and treatments that are being developed to improve the treatment of cystic fibrosis.
Our understanding of CF continues to evolve as scientists study what causes the disease and how it affects the body. These insights drive the development of new and better treatments and bring us one step closer to a cure.
Researchers, supported by the CF Foundation, have made tremendous advances to improve the health and quality of life of people with CF. We are committed to providing the tools and resources you need to continuously build upon this work.
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.
Cystic fibrosis occurs when the cystic fibrosis transmembrane conductance regulator (CFTR) protein is either not made correctly or not made at all. The goal of both gene editing and gene replacement therapy is to give the cells of people with CF the ability to make normal CFTR proteins.
For gene editing or gene therapy to work, the gene editing tools or the correct CFTR gene must get inside the cells of a person with CF. These genetic therapies are made up of engineered molecules of DNA, depending on the method used.
Right now, CF scientists are focusing on the challenge of delivering DNA to the cells of the lung, because that is one of the organs most severely affected by the disease. Getting DNA molecules into lung cells is a big challenge because of the body's natural defenses to block germs and other foreign invaders from entering cells. There are two main barriers that make it difficult to deliver DNA to lung cells:
It is important to keep in mind that genetic treatments would work only in the cells that receive the engineered DNA. If cells in the lung receive gene therapy and produce functional CFTR proteins, cells in other parts of the body still would not be able to produce CFTR. Different delivery methods would be needed to get DNA to other organs affected by CF, such as the intestine or pancreas. Although the lung is considered a priority for delivery, the Foundation also is considering approaches to deliver to other affected organs.
To treat a complicated disease like CF with genetic techniques, researchers in gene editing and gene replacement therapy need to work with researchers who are developing technologies for gene delivery. By working together, the researchers can develop the most effective genetic therapies for CF. The CF Foundation organized conferences in 2016 and 2018 to enable researchers working in each of these areas to learn about each other's work, establish relationships, and lay a foundation for new collaborations on CF therapies.
If DNA is inside a chemical package, it can enter cells much more easily. For example, DNA can be packaged inside a lipid coating to form a liposome. A liposome is a membrane that is similar to the membrane that surrounds the cell. The liposome can fuse with the membrane of the cell -- like two water droplets merging into one bigger drop -- allowing DNA from the liposome to transfer to the inside of the cell.
In 2015, a clinical trial in England tested whether DNA packaged into liposomes could deliver a gene replacement therapy for CF. The liposomes with the normal CFTR DNA were put in a nebulizer, and people with CF inhaled them once per month for a year. The research indicated that the liposomes were safe and did not cause many side effects. The participants who inhaled the liposomes also had a small improvement in forced expiratory volume (FEV1). This small change means that using liposomes to deliver a gene replacement therapy for CF is promising, but a more efficient gene delivery system will be necessary to achieve a bigger, longer-lasting improvement in lung function.1
Scientists are working on developing new coatings to make gene delivery more effective. They are studying how to make better liposomes and exploring other chemical coatings called polymers. Scientists are also figuring out how to compress DNA into small, dense structures called nanoparticles that can then be coated with lipids or other chemicals. Because nanoparticles are smaller and can be more completely coated with lipid than traditional liposomes, they may deliver DNA more effectively to cells.
Researchers also have experimented with using viruses to deliver DNA into cells. Viruses are tiny packages of DNA inside a protein coating. The protein coating can allow the DNA to get inside a cell. There are thousands of different types of viruses. Each type can deliver its DNA to only a few kinds of human cells, based on the properties of its protein coating. For example, some viruses can deliver DNA only to liver cells, and others only to white blood cells.
The genetic material that is normally found inside viruses can be harmful and cause illness. If the harmful DNA inside the virus is replaced by helpful DNA, such as a corrected gene, the virus can be used as a vehicle to get the helpful DNA into cells. Viruses that only contain helpful DNA should not cause illness.
Gene replacement therapies that use a virus to deliver DNA to cells have been tested in people with hemophilia, muscular dystrophy, metabolic disorders, and a specific type of blindness. These therapies were found to be safe and effective. Viruses have also been used to deliver DNA for a treatment for leukemia, called CAR-T therapy. This therapy was also successful and was recently approved by the U.S. Food and Drug Administration.
The main difficulty with using viruses to deliver DNA for a CF therapy is that very few viruses preferentially deliver DNA to the cells in the lung. Scientists are engineering viruses with unique protein and lipid coatings that will allow them to deliver DNA to lung cells. These new viruses are being tested in animals, and it will probably be several years before they can be tested in people with CF.
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