Week 9: Ethics of Gene Therapy

Gene therapy is the “insertion of genes into an individual’s cells and tissues to treat a disease, and hereditary diseases in which a defective, mutant allele is replaced with a functional one.” That being said, the trouble is trying to get the gene in the right place. This technology has already been used with some success, as in the case we read about in class where a young girl was born with SCID, a rare genetic disease that essentially meant she lacked a healthy immune system. This therapy allowed this young woman to ultimately lead a relatively healthy life, which would have been impossible without this therapy (she would have been relegated to live life as another “bubble baby”).

However, since human gene therapy is in its infancy and very complex, there are many issues from various perspectives (legal, biological, medical, philosophical, and religious to name a few) that need to be taken into consideration moving forward.

Also, there are currently various methods for getting the gene into the right location…including inserting a normal gene into a nonspecific location (this is most common), swapping one specific gene for another, genes can be repaired through selective reverse mutation (whatever that is!) and through ‘regulation’ (turning the gene on or off)…all of these approaches will require further rigorous development and experimentation to fully develop the procedures.

Then, there is the issue of the vectors for getting genes into place. Viruses are one of the current methods used to insert genetic materials into their ‘host’; however, the problems with this approach are many…beginning with: how do we know that the virus takes the gene to the place it need to go? Then, there is the issue of preventing undesirable effects, and insuring that the new gene won’t disrupt any of the already existent genes in the genome.

And if those weren’t enough of a red flag for consideration of this technology, there is always the specter of abuse of the power this technology wields, be it for creating ‘designer babies’ -or- using it for less than beneficial purposes, such as a weapon for military purposes. In fact, it has been suggested, that it is just this sort of genetic tinkering that created Lyme’s disease (suspected to be a genetic distortion of Syphilis, with which it shares common etiology and symptoms).

And yet, if we were that young girl, living in a bubble, who wouldn’t leap at the chance for a relatively ‘normal’ life…regardless of the potential cost? This therapy is too young at present and needs much more oversight and contemplation moving forward.