Sofie
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Sofie has Cystic Fibrosis. It has impacted her life greatly and in many ways, such as she has to do hours of physical therapy every day, and take dozens of drugs as well. It is a very unpredictable disease, and that makes it very hard on Sofie. She is 23 years old, and the average life expectancy for someone like Sofie is only 38 years old. She wants to know whether the genetic revolution that occurred 10 years ago might help deliver a long awaited treatment for her. She has turned to gene therapy in the hopes of understanding her disease better. The gene therapy she is seeking help from consists of man made copies of the healthy CFTR gene, of which hers is defected, and it is suspended in a fatty liquid. They thought the testing of the largest safe single dose will be complete in 2012 but now, it is 2013 and there isn't any real sign of it. It is especially hard to find a working medicine, because lungs are made to not take unknown substances. For Sophie, gene therapy is like a race against time. She's 23 and only expected to live to 38. She remains optimistic about the future, but the treatment she needs is still undergoing testing. She would be able to benefit from this because it will help Sofie clear up her lungs. It isn't all well for Sofie though, because her condition still gets worser by the day.