The participants who inhaled the liposomes also had a small improvement in forced expiratory volume (FEV 1). The research indicated that the liposomes were safe and did not cause many side effects. The liposomes with the normal CFTR DNA were put in a nebulizer, and people with CF inhaled them once per month for a year. In 2015, a clinical trial in England tested whether DNA packaged into liposomes could deliver a gene replacement therapy for CF. 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. A liposome is a membrane that is similar to the membrane that surrounds the cell. For example, DNA can be packaged inside a lipid coating to form a liposome. If DNA is inside a chemical package, it can enter cells much more easily. 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. By working together, the researchers can develop the most effective genetic therapies for CF. 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. Although the lung is considered a priority for delivery, the Foundation also is considering approaches to deliver to other affected organs. Different delivery methods would be needed to get DNA to other organs affected by CF, such as the intestine or pancreas. 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. It is important to keep in mind that genetic treatments would work only in the cells that receive the engineered DNA. DNA can only get through the membrane and into the cell if it is coated with a special packaging material, which scientists are still trying to develop. That means that even if the DNA gets through the mucus, it still needs a way to get through the membrane and into the cell. Cell membrane: All cells are surrounded by a membrane that keeps the contents of the cell inside and everything else outside.In the lungs of people with CF, these cells are under a much thicker layer of mucus and are even more difficult to reach. The mucus is intended to trap anything that is inhaled into the lung to make it more difficult to get to the cells. Mucus: Even in healthy lungs, lung cells are underneath a protective layer of mucus that coats the entire airway.There are two main barriers that make it difficult to deliver DNA to lung cells: 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. 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.
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