Study of Mitochondrial disease raise hope for treatment

Study of Mitochondrial disease raise hope for treatment

Scientists in the US say they have taken the first step towards treating individuals born with mitochondrial diseases-debilitating genetic disorders.

Their study, in Nature, showed there could be production of healthy cells in the laboratory from affected patients.

The team suggests future treatments could use the healthy tissues to repair the heart and other organs that are damaged.

Experts said that the study was exciting and “beautifully executed” but cautioned there was still far more work ahead.

One in every 6,500 babies has severe mitochondrial disease which leaves them lacking energy and resulting in blindness, heart failure, muscle weakness and even death.

Mitochondria are tiny compartments inside nearly every cell of the body that converts food into usable energy.

Defective mitochondria are passed down from the mother only and this year, the United Kingdom made a landmark decision to allow the creation of “three-person babies” to prevent children from being born with the fatal disease.

Anyone who currently has the condition however, cannot benefit from the measure.

Cloned Sheep

To produce healthy samples from affected patients, the team at Oregon Health and Science University used two techniques.

The first technique relied on the fact that out of the hundreds of mitochondria in every cell, there is a mixture of defective and healthy ones.

By taking and growing multiple of skin cells, the researchers were able to find a range of cells with between 0% and 100% healthy mitochondria- (follow this link for additional information).

The second technique involved the same method used to produce the first cloned animal-Dolly the sheep.

The core genetic information, the nucleus, was placed inside the egg of a healthy woman and then electricity was used to encourage the egg into developing to a healthy embryo.

Both methods produced stem cells-a type of cell that can be transformed into any other kind-which hold great promise in medicine.

“Cure on the horizon “

Prof Shoukhrat Mitalipov, one of the researchers said, ‘There is a long way to go-it’s the same issue as in regenerative medicine”

“There has been considerable research into turning stem cells into the desired tissue type.

“Then the next step is to harvest and transplant them into a patient, to ensure they implant, integrate and function well.”

This is the greatest challenge, but Prof Mitalipov says: “Today we can say that a cure is on the horizon”

Prof Robin Lovell-Badge, from the Francis Crick Institute, termed the research as “interesting and important.”

However, he warned challenges lay ahead in harnessing this approach as treatment. “The problem here will be to devise methods to replace cells within the patient.

“This might be feasible for muscle, where there are stem cells that are responsible for cell turnover within the tissue, but it will be very difficult for heart and brain cells, where cell turnover is very low or non-existent.”

Prof Darren Giffin, from the University of Kent, said: “This is clearly a very exciting study that might pave the way to possible treatment of mitochondrial disorders.

“It will, however, be some time before it can be applied clinically.”




Related Articles