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CYSTIC FIBROSIS. what is the current research on cystic fibrosis? how is this reshearch helping with finding a cure or a better understanding of the disease? how is this research being funded? what are the future prospects for finding a cure??
Question Date: 2003-03-10
Answer 1:

Here's a little background on cystic fibrosis. Basically, it's caused by a mutation in the gene that helps us to make mucus. That may just seem like it would give you a stuffy nose or something, but mucus has some important functions. It helps to keep our lungs clean by carrying small particles out of the lungs. Then we can swallow it, blow it out of our noses, etc. People with CF often have many lung infections because the mucus is so thick and sticky it stays in the lungs. The thick mucus can also block the tube that allows digestive juices to leave the pancreas and enter the intestine. Without the enzymes in this liquid, food can't be broken down enough to get much nutrition from it.

People with CF may have to take antibiotics to treat their lung infections, have people thump them on the back for long periods every day (to help clear mucus from the lungs), use inhalers, take nutritional supplements, or even be fed by tubes.

There is a lot of research going on. To find out more about specific programs and funding, check out:
cystic fibrosis

Answer 2:

The causes of CF seem to be well understood at this point - it is caused by a defective gene product called delta F508 that disrupts a protein called cystic fibrosis transmembrane conductance regulator - CFTR.

There have been efforts to use gene therapy to replace this defective gene with a"normal" copy. The problem lies with transporting the gene into the cells of the lungs (it can not be done with high efficiency by pills or injections). The current best hope is to encapsulate the DNA in very small particles so that it can be delivered by inhaling an aerosol of these "nanoparticles". Current clinical trials are underway. Funding comes from many sources, government as well as industry/pharmaceutical/biotechnology companies.

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