Scientists have managed to edit the DNA inside of a living patient for the first time ever, and it seems we are upon a new milestone in the history of science. A group of researchers from UCSF Benioff Children’s Hospital in Oakland has tried a new treatment that consists of modifying the human genome of a patient, this with the intention of curing a genetic disorder.
For several years have scientists worked at several levels in gene therapy, where they sought out to modify genes in a laboratory before re-incorporating them into a human being.
In this case, we are talking about such a modification taking place inside the patient’s body, and not in a laboratory.
Hence its relevance, since this could change forever the genetic treatment of ailments that until now were incurable.
Doctors of the future, playing ‘God’?
Brian Madeux, 44, has become part of the story, as he is the first patient to receive a type of intravenous drug, which contains billions of copies of a corrective gene that is linked to a genetic tool that is able to modify its DNA.
This tool is known as the Zinc Finger Nucleases and its the successor of CRISPR. Its job is to basically cut the DNA in the right place, as if it were a “molecular scissors”, remove the gene responsible for the condition and insert the corrective gene. The instructions for this entire complex procedure are encoded in a virus that was modified to target the patient’s liver.
The patient suffers from a rare genetic disorder called Hunter’s Syndrome, which is caused by a missing or malfunctioning enzyme.
This causes the body to not be able to break down certain carbohydrates, causing them to accumulate and cause damage, such as increased head and liver size, hearing loss, joint stiffness, difficulty breathing and even mental problems.
Unfortunately, today there is no cure and its treatment consist of enzymatic therapies every week.
Most Hunter Syndrome patients die between 10 and 20 years, so Madeux’s case is one of those rare exceptions.
Therefore, he decided to accept this experimental treatment knowing that there are many things that could go wrong.
Madeux says it’s worth giving it a chance despite the risks, as this could change the lives of millions of people, especially children, who are the most affected by this type of disease.
Time to wait
Scientists say that the next thing to do is wait for at least three months, which is when they will get the results of the genetic modification and they will know if it was done correctly and if or not there is collateral damage.
In case the treatment proves successful, the team of scientists will extend their tests to new adult patients to continue improving the technique, aiming to start the treatment in children in the near future.