Genetically engineered stem cell treatment for Parkinson's restores movement in rats

Publicly released:
Australia; International
Photo by Jaime Maldonado on Unsplash
Photo by Jaime Maldonado on Unsplash

An international research team has genetically engineered stem cells to improve a potential treatment for Parkinson's, restoring movement in rats. Lead researcher Mark Denham, originally from Australia, says stem cells are a promising treatment for Parkinson's as they can transform into specific nerve cells needed to treat the condition, however many generate the wrong type of nerve cells. He says the researchers tested a way of genetically engineering the stem cells to produce the right nerve cells more often, and the treatment was successfully tested on rats. The researchers say generating these nerve cells with higher accuracy means that should this treatment progress and be used on humans, it could result in longer-lasting treatment effects with shorter treatment duration.

Media release

From: Springer Nature

Improving the treatment of Parkinson's patients

The future treatment of Parkinson’s Disease has undergone tremendous development in recent years. Now, an advancement in research has emerged, delivering the strongest results for both side-effect-free and long-lasting treatment effects.

The results are just published in Nature Communications under the title "Enhanced production of mesencephalic dopaminergic neurons from lineage-restricted human undifferentiated stem cells."

In the new research findings, DANDRITE group leader and Associate Professor Mark Denham has developed a method that ensures much higher purity of the so-called dopamine cells, which are crucial in connection with Parkinson's disease.

"Stem cells offer promising potential for treating Parkinson's disease by transforming into specific nerve cells. However, the precision of this transformation poses a significant challenge with current methods, resulting in low purity," Mark explains.

Achieving high purity is critical for effectively restoring movement in patients.

In the Denham Lab, stem cells were genetically engineered to prevent them from generating the incorrect types of nerve cells. The newly engineered stem cells have an enhanced ability to produce the specific nerve cells required for Parkinson's treatment known as the dopaminergic cells.

Furthermore, the researchers show that the genetically engineered stem cells led to the restoration of movement in animal models. This advancement is a potential new therapeutic approach for treating Parkinson's disease patients.

Experiments on rats have shown that both the quantity and purity of cultured stem cells are critical for the number and duration of treatments.

“Using our genetically engineered cells we generate a higher purity of dopamine cells, for patients this will reduce the recovery time and diminish the risk of relapse and medication use. My goal is to help patients stay off their medication, which requires high purity. So, my next step is to transfer my method to clinical trials,” Mark states

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Journal/
conference:
Nature Communications
Research:Paper
Organisation/s: Danish Research Institute of Translational Neuroscience (DANDRITE), Denmark
Funder: Funding: This study was supported by Lundbeckfonden (grant no. DANDRITE-R248-2016-2518 (M.D., A.N.) and R248-2017–431 (A.N.)), the Parkinsonforeningen (M.D.), and The Danish National Research Foundation grant no. DNRF133 (A.N.). M.D. is a partner of BrainStem—Stem Cell Center of Excellence in Neurology, funded by Innovation Fund Denmark.
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