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