Progress in gene therapy treatment for Parkinson's

Researchers, funded by Parkinson's UK, have produced positive results in mice for a gene therapy treatment that directly targets the production of the alpha-synuclein protein.

The study, due to appear in the December Issue of the scientific journal Molecular Therapy, highlights the potential of packaging tiny gene therapy molecules in minicircles to help deliver the treatment to the brain.The treatment targets a toxic protein involved in Parkinson’s, alpha-synuclein. 

What is alpha-synuclein? 

While normal alpha-synuclein protein is thought to help brain cells communicate, one or more toxic forms of this protein are linked to Parkinson’s. Recent research also suggests that the protein is responsible for the spread of Parkinson's from cell to cell. As such, there’s a lot of interest in targeting alpha-synuclein to slow or stop the progression of Parkinson’s. 

How could gene therapy help? 

The idea is that gene therapy could provide the genetic instructions that cells need — to replace a faulty gene with a functional one or provide instructions for switching off the production of misbehaving proteins like alpha-synuclein.
However, getting the genetic instructions from the lab into brain cells is tricky. Right now, the most effective way of doing this is to ‘hijack’ a virus but this research investigates an alternative method. 

Towards a long term therapy

In this project, the researchers have been working on how to package and deliver a type of gene therapy that will switch off the production of alpha-synuclein. The researchers used a molecule called shRNA which works for long periods of time, making the therapy suitable for treating long-term neurodegenerative conditions.
The team found that by making the gene therapy instructions into minicircles - microscopic circular pieces of DNA with no unnecessary packaging - they were able to reduce the buildup of toxic alpha-synuclein to protect brain cells and improve symptoms in a mouse model of Parkinson's.
Dr Beckie Port, Research Communications Manager at Parkinson’s UK, said:

"Parkinson's affects around 1 in 36 of us in our lifetime but, to date, there is no cure and no treatment that has been proven to slow down the gradual loss of precious brain cells.
"There is much interest in gene therapies to target problematic proteins in neurodegenerative conditions. In Parkinson's, this protein is alpha-synuclein and treatments that target this protein holds much potential to slow or even stop its progression.
"While much of the work is still in the early stages, there have been a number of promising publications in the last few years that build hope that we could be on the path to a treatment that could have a huge impact for everyone with Parkinson's."

Gene therapy and Parkinson's

Find out more about gene therapy and how it is being investigated as a potential treatment for Parkinson's.