What is Cystic Fibrosis?
Cystic Fibrosis (CF) is a hereditary disorder that affects
the cells of the lungs, pancreas, and small intestines. This disease causes
abnormal amounts of mucus to be secreted from these regions and usually results
in coughing, poor digestion, and extremely higher risks of infection. If a
patient goes untreated they will most likely develop lung infections that are
fatal and more likely than not will not live past their late 20’s. Cells that
are within the lung’s lining produce a lot more mucus because of a mutation
found on chromosome 7 that is rightfully called the Cystic Fibrosis
Transmembrane Regulator (CFTR) gene. This mutation is a deletion of an amino
acid that completely changes the overall function of the gene.
Photo courtesy of the NIH
How can Gene Therapy help?
Cystic Fibrosis appears to be an excellent candidate for
gene therapy because adding the gene that was deleted would most definitely fix
the mucus production of the cells. So how do we do it? The first step is to
find a vector to carry the normal gene into the body. By injecting the gene
into a virus, the virus will be able to “infect” the cells with the correct
gene and help control the mucus production.
Let’s do it!
This idea of gene therapy sounds so easy to accomplish,
however, like anything else, it requires a lot of funding. Currently, there are
patients and scientists undergoing clinical trials for this research but it is
going much slower than anticipated. The idea of gene therapy is looking
extremely promising and seems like a beneficial outcome of genetic engineering.
References:
It's really amazing how the scientists used the virus to correct genes by injecting them with the replacement gene. I would like to read more about it and how the Genetic Engineering can help us in too many different ways. thank you Bridget for bringing this up because 'Genetic Engineering' got my interest.
ReplyDeleteI guess that we can treat many other diseases by using gene therapy. The idea of gene therapy sounds very cool and fast to treat diseases, however; I think that we need to do more studies on gene therapy just to avoid unexpected results. I'm excited to learn more about gene therapy and genetic engineering.
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