London - A cure for “horrendous” childhood illnesses is on the horizon, scientists said.
They made the prediction after using a cloning technique to create a repair kit for brains, muscles and hearts ravaged by mitochondrial disease.
This group of genetic illnesses can trigger miscarriages and stillbirths.Other children die in infancy or become progressively more ill as they enter adulthood and there is, as yet, no cure.
But the US breakthrough also raises the spectre of babies being cloned to order in labs. The scientists stress their aim is to provide a treatment for seriously ill children. But there will be fears a rogue scientist will copy their work to try to clone humans. In the study, scientists from Oregon Health and Science University started by taking slivers of skin from adults and children with mitochondrial disease.
They then used two techniques to turn these into stem cells – master cells capable of transforming into any of the cells in the body. The most successful of the two techniques was also the most controversial. Using a process similar to that used to clone Dolly the sheep, they took the skin cells and put them in hollowed-out eggs.
They zapped the eggs with electricity to make them start developing into embryos. When the cloned embyros were five or six days old, and the size of a pinhead, they were mined for stem cells. These master cells were then turned into brain, muscle and heart cells.
Crucially, the cloning process eliminated the mitochondrial disease – meaning the resulting cells were healthy. A perfect match for the patient who donated the skin, they could potentially be used to fix organs devastated by disease.
Researcher Shoukhrat Mitalipov said: “To families with a loved one born with mitochondrial disease waiting for a cure, today we can say that a cure is on the horizon.”
Professor Darren Griffin, of the University of Kent, said: “These diseases can be horrendous and any routes to therapy are most welcome.” Professor Robin Lovell-Badge, a stem cell expert at the Francis Crick Institute in London, said using the cells to cure mitochondrial disease was “wishful thinking”. Others fear maverick scientists will exploit the technique to try to give grieving parents a replacement for their dead child. Dr Peter Saunders, of the Christian Medical Fellowship, called the cloning cure “highly suspect ethically”.
And Dr David King, of campaign group Human Genetics Alert, said using the technique to treat mitochondrial disease could give full-on human cloning a new legitimacy. But Professor Lovell-Badge said it is so difficult and dangerous “no one in their right mind would do it.”
Mitochondrial disease hit the headlines earlier this year when Parliament approved an IVF technique that would allow couples blighted by it to have healthy children. This is controversial as any babies that are created will effectively have three parents – two mothers and a father.
Daily Mail