For the first time in Europe, humans will be genetically modified after the regulators have given the go-ahead to trial DNA-splicing therapy.
The destructive blood disorder called beta thalassaemia (which reduces the production of haemoglobin) could be cured with this therapy.
Haemoglobin is the carrier of the oxygen throughout the body, and without sufficient amounts, those with the disease can be left with anaemia, slow growth, fatigue and shortness of breath.
The scientists at the biotech company Crispr are hoping that they will be able to alter the body’s code to stop the genetic mutation and restore the healthy levels of haemoglobin.
It is the first time for the disease to be treated with this method in Europe, and the trials are said to be promising.
Similar trials have been made in China because the restrictive regulations are not the same as in Europe or the U.S.
Professor Robin Lovell-Badge from Group Leader at London’s Francis Crick Institute says that they will look back and think that this was the real beginning of gene therapy.
For the past 30 years, this type of therapy had been used by doctors to dispense the missing DNA from damaged cells, increasing their effectiveness, but Crispr’s work may be a more long-term solution and it can also prove to be cheaper.
The therapy uses the natural defense mechanism of bacteria, which is carrying strands of deadly viruses so that they can recognize them.
If the bacteria come into contact with the virus, it will be able to release an enzyme in order to attack it and cut away at that form of code.
Scientists have created their own cutting mechanism which removes mutated areas of DNA, but, however, even though this treatment will hopefully be used one day on humans, the trials will take place outside of the human body.
Stem cells will only be harvested from the body, but grown in a laboratory before their level of foetal haemoglobin is increased.
It is the high level of protein which is present in babies, but it is later repressed when the human reaches adulthood.
The plan is to remove the gene which represses the growth of protein, allowing the patient’s bone marrow to again produce high levels of haemoglobin.
Darren Griffth, Professor of genetics at the University of Kent says that everything he has seen suggests that it’s very safe and effective and that he believes that the trial results will be positive.
The University of Pennsylvania has enlisted for a trial which plans to use this method to treat patients with cancer.