Chapter 2: Gene Therapy and Cystic Fibrosis

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:

http://www.ncbi.nlm.nih.gov/pubmed/22229571

http://learn.genetics.utah.edu/content/genetherapy/casestudy/

http://www.nhlbi.nih.gov/sites/www.nhlbi.nih.gov/files/images_252 

Chapter 1: An Introduction

Ever since the Human Genome Project, science and researchers have been increasingly more and more fascinated by genetics and the DNA that makes us who we are. Genes influence traits and behaviors as well as health and disease and although the science community has learned a lot, there is so much left for the science community to learn about what makes people who they are. The Human Genome Project discovered that there are approximately 20,500 human genes. This completed sequence allows us to identify the locations of genes and the overall structure and organization of our genome. This knowledge has set a basis for what research we are doing on genes in general.

 

The Cons to this Technology

When most people think about Genetic Engineering, the idea of “Designer Babies” always appears to come up as a topic. From a parent’s perspective, it would seem incredible if we were able to remove a genetically linked disease from our child’s embryo. Unfortunately, this leads to the question of where does this genetic-tweaking end? What are the moral/ethical implications of making sure that our child visually looks adequate according to society? It also questions the child’s lack of autonomy for these decisions because they are decided at such a young age. Many people argue that this idea of “Designer Babies” is just the beginning stages of eugenics and we should stop research.

Photo courtesy of University of London Forum on Genetic Engineering

The Pros to this Technology

Although there would seem to be a lot of downfalls for this technology there are actually a lot of possible positive outcomes of such research. One thing in particular is gene therapy. Gene therapy is a technique that is being used as a treatment as opposed to drugs or medication. By using this therapy doctors and scientists are allowed to treat a disorder by way of inserting a gene into a patient’s cells. As of right now, the treatment is still under study but appears to show a lot of promise in terms of treating inherited disorders, types of cancers and some viral infections.


Although there is still so much to learn in this field of Human Genetic Engineering, there appear to be a lot of promising research that will hopefully benefit science and healthcare treatments in the long run. Overall, the subject needs a lot more research done and we are already making a decent amount of progress.



References:

http://www.nature.com/scitable/topicpage/genetic-inequality-human-genetic-engineering-768

http://www.genome.gov/12011238

http://ghr.nlm.nih.gov/handbook/therapy/genetherapy

 

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