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Gene editing is considered by many as the future of medicine. The potential uses attributed to the field range from curing all kinds of diseases to making humans practically invulnerable. For the first time, scientists have managed to conduct gene editing inside an actual living human in an attempt to cure a rare genetic disease. If successful, this could lead to a new generation of medical marvels.
The experiment was conducted in Oakland, California on November 13th, where doctors performed the gene editing process on a 44-year-old male, Futurism reports. The patient was apparently suffering from a condition known as Hunter syndrome. An exceedingly rare disease, it involves a missing enzyme that is essential for important bodily functions.
Without the enzyme, the body suffers damage that inhibits mental growth. Doctors basically tried to replace the defective parts of the gene responsible for this and replace it with functioning ones. It’s a bit like replacing parts of a car’s engine when it breaks.
This particular treatment was developed by the biotechnology firm that’s based in California called Sangamo Therapeutics. In an interview with The Scientist, the company’s CEO, Sandy Macrae said that it was a “privilege and a responsibility” to be involved in the first gene editing treatment of its kind.
Now, it’s worth noting that the gene editing process that the scientists in this case used was not CRISPR Cas-9. Rather, it was zinc finger nucleuses (ZFNs). Apparently, this method is more difficult than CRISPR because it’s more unwieldy, but it comes with superior precision.
In any case, if this treatment proves successful, it could open the door to many other treatments of several other genetic diseases. When medicine could only manage the symptoms of rare genetic disorders before, patients can actually hope for a real cure. Of course, the level of precision and success rate would still need to increase.
