The future of gene therapy in Parkinson’s disease

At this stage, brain diseases such as Parkinson’s disease have lots of treatment that improve symptoms and quality of life of patients, but no therapy that fixes the underlying problem. This is clearly something important to search for.

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An interesting but very early study, in other words far from being tested in humans is showing promise. In the past, researchers have used viruses to try and bring in new genetic material into the body to improve a condition. Now, researchers are using the body’s own immune cells to deliver a chemical that may help the brain heal and may even heal itself.

The researchers genetically modified white blood cells called macrophages to produce “glial cell-derived neurotrophic factor”, or GDNF, and then also to be able deliver it to the brain of a mouse model of Parkinson’s disease. Glial cells provide support and protection for nerve cells throughout the brain and body, and GDNF can heal and stimulate the growth of damaged neurons. They showed this was possible, and new research will continue to see how far this technique can go.

Reference:
Yuling Zhao, Matthew J. Haney, Richa Gupta, John P. Bohnsack, Zhijian He, Alexander V. Kabanov, Elena V. Batrakova. GDNF-Transfected Macrophages Produce Potent Neuroprotective Effects in Parkinson’s Disease Mouse Model. PLoS ONE, 2014; 9 (9): e106867 DOI: 10.1371/journal.pone.0106867

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