First ever attempt to edit genes inside human body carried out in the US

Doctors in the US have made the first ever attempt to edit genes within the human body on Monday. The experiment to alter the DNA was conducted on a patient who was suffering from Hunter’s syndrome.

Doctors at UCSF Benioff Children's Hospital in Oakland, California administered the intravenous treatment(IV) that will provide the body of the patient Brian Madeux with instructions to change the DNA, said a report by the Telegraph.

Madeux’s body, which is suffering from Hunter’s syndrome is unable to break sugary molecules called mucopolysaccharides leading to the built up of molecules inside the body. This ultimately leads to brain and organ damage and in some cases, even death.

While in usual cases patients suffering from the problem are given enzyme replacement therapy on a regular basis to break down the molecules, Madeux hailing from Arizona reportedly decided to undergo the trial as he is suffering from ‘pain every second of the day’.

Through the IV treatment, patient’s body will receive viruses that are altered to carry instructions to the DNA. These will then travel to the liver where, according to the report, the cells will ‘use the instructions to make molecular scissors called zinc finger nucleases and prepare the corrective gene’.  The newly corrected genes will then cut the patient's DNA at precise spots. If the experiment succeeds, the new genes will give instructions to the cells to make the enzyme that is needed to break the molecules.

While the procedure to correct the DNA has been carried out earlier this was all done after the cells were taken out of the body.

The news of the experiment has led to varying reactions from different corners. While Dr David R. Liu, from Harvard University, supported the experiment and was quoted saying in the report as, “The Hunter's syndrome trial, led by a pioneering company, is another reminder that we have entered an era of genome editing."

Dr Robin Lovell-Badge of the Francis Crick Institute said that it will be a challenge to make the treatment work on patients suffering other problems that are much more complex. The procedure will also be impossible to do on cells within key organs like brain, heart and liver.

But for the time being, the medical community is waiting eagerly to know the result of the experiment carried out on Madeux. If successful, many more patients suffering from various ailments could benefit from it.