Scientists use gene editing to create 'tougher' stem cells

Researchers at the University of Edinburgh have used cutting-edge gene-editing techniques to create stem cells that are resistant to developing Parkinson's.

Stem cells are special cells which can develop into almost any cell in the body. They hold huge hope for Parkinson's because they could be used to create healthy new brain cells to replace those lost in the condition.

Major progress has already been made towards stem cell therapies but there are also significant challenges.

Previous research has suggested that new healthy brain cells transplanted into the Parkinson’s brain may gradually develop the condition themselves. 

Researchers believe this is due to a protein called 'alpha-synuclein' invading healthy cells and triggering the formation of sticky clumps called Lewy bodies.

What the team did

In this research, the team used pioneering gene editing techniques to snip out the alpha-synuclein gene from their stem cells.

In lab tests, the stem cells were transformed into brain cells that produce dopamine – a key brain chemical that is lost in Parkinson’s – in a dish. The cells were then treated with a chemical agent to induce Lewy bodies.

Cells that had been gene-edited did not form the toxic clumps, compared with unedited cells, which developed signs of Parkinson’s.

Professor David Dexter, our Deputy Research Director, said:

“This pioneering work has the potential to deliver long-term, life-changing treatments for people with Parkinson’s.

“This should mean that the Parkinson’s stem cell transplantation trials stand a significantly better chance of providing long term benefits for people who are suitable for this kind of treatment.”

The study, published in the European Journal of Neuroscience

More on stem cell research

There are already clinical trials underway across the world investigating the potential benefits of stem cell therapies for Parkinson's.