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Three parent babies needless as 'cure' for mitochondrial disease on horizon

posted 25 Jul 2015, 21:41 by Crossways Dorking   [ updated 10 Sep 2015, 03:10 ]
Damaged cells can be turned back into their original stem cell form where they no longer have mutations


The creation of three parent babies may no longer be necessary after scientists discovered a potential cure for mitochondrial diseases like muscular dystrophy.

In February the government changed the law to allow IVF clinics to use donor DNA from a 'second mother' to repair the defective eggs of women at risk of passing on mitochondrial disease to their children.

It is controversial because it would result in babies having DNA from three people for the first time in human history.

But a new study from Oregon Health & Science University suggests that using genetic material from a third person may not be necessary after all. They have discovered that even people suffering from mitochondrial disease can still produce healthy mitochondrial DNA which can be used to mend the defects.

Mitochondria act like batteries, powering cells. But in mitochondrial disease the DNA does not fuel cells fully, causing problems such as muscle wasting, as in the case of muscular dystrophy.


Dr Shoukhrat Mitalipov, of the Centre for Embryonic Cell and Gene Therapy, has shown that it is possible to turn damaged cells back into their original stem cell form where they no longer have mutations. Those cells can then be multiplied in a lab and transplanted back into the body to repair damaged tissue.

“To families with a loved one born with a mitochondrial disease waiting for a cure, today we can say that a cure is on the horizon,” said Dr Mitalipov

“Over the past several years, we have been working to generate stem cells for use in combating disease.

“This critical first step toward treating these diseases using gene therapy will put us on the path to curing them.

“And, unlike unmatched tissue or organ donations, combined gene and cell therapy will allow us to create the patients’ own healthy tissue that will not be rejected by their bodies.”

Mitochondrial mutations can cause a vast range of fatal or severely debilitating diseases, including diabetes, deafness, eye disorders, gastrointestinal disorders, heart disease, dementia, and several other neurological diseases.

The researchers discovered that when skin cells from patients with mitochondrial disease are converted back to stem cells in a lab some of them were free from mutations. Those corrected stem cells can then be multiplied and transplanted back into the body. Although the work has only been done in the lab so far, the team is hopeful it could soon be tested in patients who have few treatment options available.

The breakthough was welcomed by British scientists as ‘exciting’ but they warned it could take some time to set up clinical trials.

Prof Darren Griffin, Professor of Genetics and Director of the Centre for Interdisciplinary Studies of Reproduction (CISoR), University of Kent, said: “This is clearly a very exciting study that might pave the way to possible treatment of mitochondrial disorders.

“These diseases can be horrendous and any routes to therapy (including palliative care) is most welcome.

“The authors have devised a very elegant system to correct the genetic defect in question. It will however be some time before it can be applied clinically given the need for clinical trials etc.”

Dr Dusko Ilic, Reader in Stem Cell Science, King’s College London, said the study was ‘beautifully executed’ and could eventually allow mothers at risk of passing on mitochondrial disease to use their own healthy mitochondria to repair their eggs.

“Following this approach, theoretically, one might be able to generate eggs with all healthy mitochondria from the mitochondrial disease carrier and in such a way eliminate a need for the healthy donor mitochondria or "the third parent",” he said.

The research was published in the journal Nature.


Source: http://www.telegraph.co.uk/news/science/science-news/11741788/Three-parent-babies-needless-as-cure-for-mitochondrial-disease-on-horizon.html






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