Humans will be genetically modified for the first time in Europe after regulators have given the go ahead to trial DNA-splicing therapy.A destructive blood disorder known as beta thalassaemia, which reduces the production of haemoglobin, could be cured using this therapy.Haemoglobin carries the oxygen the body needs to its cells and without sufficient amounts those with the disease can be left with bone deformities, anaemia, slow growth, fatigue and shortness of breath.
Scientists at the biotech company Crispr hope that they can alter the body’s code to stop the genetic mutation and restore healthy levels of haemoglobin.A destructive blood disorder known as beta thalassaemia, which reduces the production of haemoglobin, could be cured using a new type of therapy.A destructive blood disorder known as beta thalassaemia, which reduces the production of haemoglobin, could be cured using a new type of therapy
The disease is the first to be treated using this method in Europe and experts have said that the trials hold promise.Similar trials have taken place in China however they do not have the same restrictive regulations as Europe or the U.S.Professor Robin Lovell-Badge, Group Leader at London’s Francis Crick Institute, told the Sunday Telegraph UK: “We will look back and think that this is the real beginning of gene therapy.”
This type of therapy has been used for the past 30 years as doctors dispense the missing DNA from damaged cells to increase their effectiveness.But the work taking place at Crispr may be a more long-term solution which has also proven to be cheaper.The therapy uses bacteria’s natural defence mechanism, which is carrying strands of deadly viruses so that they can recognise them.If they come into contact with the virus they are able to release an enzyme in order to attack it and cut away at that form of code.
Scientists have taken this on board and created their own cutting mechanism, which removes mutated areas of DNA.However although this treatment will hopefully one day be used on humans the trials will take place outside of the human body.Stem cells will be harvested from the body and grown in a laboratory before increasing their level of foetal haemoglobin.This is the high level of protein, which is present in babies however it is repressed when a human reaches adulthood.
The scientists plan to remove the gene, which represses the growth of the protein and will allow the patient’s bone marrow to again produce high levels of haemoglobin.
Darren Griffth, professor of genetics at the University of Kent, told the Sunday Telegraph UK: “Everything I have seen suggests it’s very safe and effective. I think the [trial] results will be positive. And then we will be able to say, this is where it all began.”
The University of Pennsylvania has begun enlisting for a trial which plans to use Crispr to treat patients with cancer. Also, controversial plans to grow human organs inside the bodies of animals have moved one step closer to going ahead in Japan.Government officials in the Asian country are expected to overturn their current ban on the practice by the autumn, according to local reports.
An expert panel, commissioned by Japanese ministers, concluded that allowing the experiments could lead to major scientific breakthroughs.The practice, which has prompted furious backlash from animal rights campaigners in recent years, is already allowed for research purposes in the UK and US.Some Japanese biologists have left the country to pursue experiments, which critics consider ‘gruesome’, across the Pacific Ocean because of the ban.
However, The Ministry of Education, Culture, Sports, Science and Technology has consulted advice from a panel of scientific experts in relation to the practice.The panel agreed that researchers should be able to implant animal embryos with human cells into an animal’s womb, The Japan News reports.Currently, Japanese experts are only allowed to inject human induced pluripotent stem cells – which can grow into anything – into an animal embryo.
These embryos are genetically modified already to be incapable of growing certain organs using DNA editing technology Crispr.If the plans go ahead, when the Ministry reviews its current guidelines, researchers will for the first time be allowed to implement their lab work into animals.
Some scientists believe that creating human organs in animals, such as pigs, could stem the growing shortage of organs needed for transplants.On average, 20 people die each day while waiting for an organ transplant, according to the United Network for Organ Sharing. But animal welfare campaigners warn the experiments to grow human organs inside animals echo the fictional horror story ‘Never Let Me Go’.
In the dystopian novel, adapted for the hit 2010 movie, a group of English children are cloned so that as young adults their organs can be used for transplants.Real-life research has seen scientists inject human stem cells into pig embryos to produce ‘chimeric’ embryos that are part pig, part human.
Researchers at Meiji and Kyoto Prefectural universities announced they developed pigs with genetically modified organs for human transplants last month.They are reportedly the first animals developed for xenotransplantation, in which animal organs and tissues are successfully transplanted into humans.
Professor Hiromitsu Nakauchi, a geneticist at Stanford University in California, hopes to create a human pancreas inside a pig, if the ban in Japan is lifted.He and fellow scientists have already reversed diabetes in mice by giving them an organ grown in a different species – rats.Professor Nakauchi moved from the University of Tokyo to Stanford to work on the experimental project, which used iPSCs.
The results, published in January 2017, highlighted the first time an inter-species organ transplant successfully treated a medical condition.Professor Nakauchi reported the results of his scientific projects nine years ago, before they were ever confirmed to be successful. He said at the time, if the method was ever replicated using human stem cells, the pancreas of diabetic patients could be replaced via this technique.
Scientists already have to follow strict safety and health guidelines when undergoing artificial reproductive technologies.Pigs have to be raised in a clean, protected environment and to be tested for up to 40 kinds of viruses to prevent infections and make sure patients are safe.
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