Tuesday, May 17, 2011

Answers Day, Part II

"If you undergo gene therapy how many genes do you have to change in order to get the end result? All of them? One of them? I honestly do not understand how this works." - Chompy (the dog)

The gene is the basic unit of heredity, and your genes contain specific sequences of instructions for creating the various proteins required to live. If a particular set of genes in your body is defective, either because you bought them at a yard sale or your mother was your sister, you end up with a genetic disorder. Gene therapy is the process of introducing fit genes that have been working out into your body, in the hopes that they replace defective genes, repair them, or counteract their effects.

The case below is taken directly from the mixed-up files of the National Institute of Health, and should provide an easy-to-follow example of how gene therapy works:

  • The test subject is a twenty-eight-year-old male living in West Chester, Pennsylvania. Because of HIPAA concerns, we will call him "Gene".

  • "Gene" is a second-generation immigrant. His mother was born in Beers and his father came from Gouda, and the pair left the Netherlands in 1976. "Gene" was born shortly after.

  • For all intents and purposes, "Gene" should be DUTCH. However, a rare genetic disorder has turned "Gene" into a DOUCHE, and as such, he is unable to function well in everyday society.

  • "Gene" participates in a clinical study, where scientists extract some of the DOUCHE and put it in a cup. They can study the DOUCHE to see what it has in common with the DUTCH, and create a special set of instructions that will specifically target the defective genes and try to correct the shortcomings. We call these repair instructions NIEUW DUTCH, or more colloquially, New York City.

  • Scientists then engineer a virus that cannot reproduce on its own, but is hardy enough to deliver New York City into the heart of DOUCHEland without triggering a response from the immune system. The virus here does not do any work other than delivery -- it is just a reliable, braindead carrier, comparable to UPS 2-Day Service.

  • "Gene" is injected with a dose of New York City. The hope is that the repair instructions will convert every DOUCHE into DUTCH. In other forms of gene therapy, New York City would simply reproduce faster than DOUCHE, until the DOUCHE is minimized, or New York City might just generate ANTI-DOUCHE in counterbalance. The end result in all successful cases is that the genetic disorder is corrected or minimized.

  • The open issue in all of these approaches is that it takes a lot of hard work and repeat applications to deDOUCHE something. A single injection, if it even works at all, cannot immediately fix every set of defective genes. "Gene" could wake up tomorrow and be just as DOUCHEy as ever, or his immune system could recognize that he has always inherently been a DOUCHE and completely reject the foreigners and their meddling ways. Even worse, the problem could be exacerbated, and "Gene" could end up a prime example of DOUCHÉ.

Any questions?

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