IEEE Future Directions
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Gene therapy is the holy grail of genetics. From the moment the code of life was discovered, and further more when the human genome was sequenced scientists have looked into ways of correcting and overcoming the effects of the 6,000 known diseases that are genetically based.
Genetical modification can be pursued at the human egg and spermatozoa, before fertilisation occurs or immediately after on the fertilised egg. It can also be attempted by modifying cells taken from a person and then re-injecting them in the body. There have been a number of trials in both, with some promising success although we are still in the very early stages.
For most of the people affected by a genetic disorder, however, the hope would be to have a way to change the genome in their cells. This seemed like a dream but now it is becoming a matter of science.
At least this is my feeling when reading the news on the first attempt to modify genes inside cells of a person.
Researchers in the US have perfected a procedure, similar to the CRISPR/Cas 9 but based on a different tool, zinc finger nucleases, that uses a virus as a vector to reach cells, infect them and change their DNA. The Sangamo Therapeutics company has produced the modified viruses, billions of them, that have been injected in the liver of a person affected by the Hunter syndrome, a genetic diseases that hampers the breaking down of complex molecules by the liver with the result of their accumulation in the body leading to severe and -over the years- fatal outcome.
The patient has been informed of the many uncertainties tied to gene modification and has agreed to be the first in the world to try this cure. It will take three months to see if it worked… Indeed it is a completely unexplored path. The problem is that a gene plays many different roles and we are not sure we understand them all. Fixing a gene to cure a disease may actually create problems in other areas since that “faulty” gene might actually serve in conjunction with other genes to perform needed functions. Correcting the gene to what it is supposed to be may disrupt its working with other genes (these latter could be modified as result of the changes in the faulty genes so that when you restore the faulty gene you area actually breaking a chain serving other purposes).
Although fraught with question marks this evolution is very promising bringing concrete hopes to cure genetic diseases for the first time in humankind history.
