Gene editing therapy

For the first time in medical history, scientists have attempted to edit a gene inside the body in an attempt to permanently change an individual’s DNA. This in turn could cure a myriad of different diseases.

The experiment was performed on a 44 year old male in California on Monday. Researchers flooded his body with billions of copies of a corrective gene and then used a genetic tool to cut his DNA in the required spot.

The patient, Brian Madeux suffered from a metabolic disease called Hunter syndrome. Speaking of the surgery, Madeux stated that it was “humbling” to be the first person to have this kind of procedure.

There is no guarantee of course if the procedure will work or not. The results and consequences of the procedure will be known within a few months. The procedure is extremely risky, success could be life changing.

While scientists have altered people’s genes before, these were always done externally. Thus scientists would always extract the cell, manipulate them in the lab, and then return them to the patient. There have also been a number of gene therapies which do not require any kind of DNA editing. However, this is the first time that the genes have been altered directly in the subject.

The older gene therapies had lower chances of success. However, since the new editing method takes place directly within the body, chances are that it could result in a complete path breaking cure.

A researcher in the team, Dr. Sandy Macare, stated that the procedure could be likened to “Invisible mending”. However, all researchers agree that there are a number of substantial risks involved with the procedure. Thus while animal tests have been very reassuring, and while researchers have taken a number of precautions to safeguard the patient’s health- in the end, there are no certainties.

This did not bother the therapy’s first patient. In fact Madeux signed on for the procedure although he was well aware of the underlying risks. He stated that he hoped the experiment on his body would result in a procedure that would help many others to come.