There were no serious adverse reactions or complications questioning the safety of the procedure for the patient who had been the first to carry out genetic engineering in the world a few months ago by US doctors who have since been treated with a second patient. The 44-year-old Brian Madeux was the first person in the world to handle gene manipulation.
In November last year, doctors made the mistake of Hunter’s syndrome on the Arizona male DNA. This metabolic disorder is very rare, a type of mucopolysaccharidosis: those suffering from the disease are born without the genetic instructions for the production of the enzymes necessary for the decomposition of long sugar molecules. These molecules, mucopolysaccharides do not break down due to the enzyme’s failure, but accumulate in the organs and damage them. During treatment, experts in the bloodstream of Madeux received two so-called “molecular scissors”, which cut the DNA in two precisely defined places. Therapy has been developed so that “scissors” do not start unless they are in the patient’s hepatocytes.
“Madeux is fine and we have been given permission to involve a second patient who is also well-responsive,” said Paul Harmatz, a San Francisco University of California (UCSF) physician at Benioff Children’s Hospital in Oaks, who treated both patients with the same illness. Dr. Harmatz reported on a medical conference in San Diego that in the first six weeks following the intervention in Madeux, the results of the safety of the procedure were obtained. Sangamo Therapeutics, California, a manufacturer of tools for gene conversion, is testing the device for the treatment of two metabolic diseases and haemophilia (bleeding). According to the company, the additional safety performance and efficacy results are expected in the middle of the year.
Madeux had fought for four days with dizziness, cold sweats and weakness after surgery, but the symptoms eventually disappeared. He also coughed heavily, and his lungs were partially collapsed, but they were not related to gene therapy because the patient had previously struggled with similar problems. According to Harmatz, one of the most important things is that the man does not have symptoms of liver damage. This was a “serious concern” for doctors, as changes in the liver may indicate that the immune system was confronted with treatment, possibly undermining its effectiveness.
Source: erdekesvilag.hu/ Picture: m.ipon.hu /