丁香园AV

Researchers are a step closer to understanding how Parkinson鈥檚 disease develops and progresses thanks to chemical biological tools developed at 丁香园AV.

New research, , and spearheaded by Drs. Matthew Deen and Yanping Zhu, outlines new technology and methods to measure the activity of lysosomal glucocerebrosidase (GCase), an enzyme that is commonly linked to Parkinson鈥檚 disease.

Parkinson鈥檚 is the second most common neurodegenerative disease and can cause symptoms including tremors, muscle stiffness, and impaired balance and coordination that worsen over time. While the exact causes of Parkinson鈥檚 are not fully understood, low GCase activity is known to contribute to the disease and a better understanding of this enzyme could lead to improved diagnostics and treatment.

The team鈥檚 findings show that the activity of this enzyme in patients is similar in both blood cells and brain cells, which could potentially enable researchers and medical professionals to monitor how Parkinson鈥檚 progresses in the brain using blood samples.

鈥淭his is the first approach that has been shown to reliably and accurately report on the activity of this enzyme directly within lysosomes of living cells,鈥� says Chemistry and Molecular Biology and Biochemistry professor David Vocadlo, one of the co-authors of the study. 鈥淏eing able to measure the lysosomal activity of this enzyme in an accurate way could be very helpful in understanding the root causes of Parkinson鈥檚 as well as potentially helping to diagnose or track its progression.鈥�

In addition to improved diagnosis and monitoring of Parkinson鈥檚 disease, the researchers hope that their work will help in the development of new drugs. 鈥淯ltimately these tools could be used in clinical trials to assess the efficacy of strategies aiming to increase the activity of GCase in patients,鈥� Vocadlo says, noting that their approach is already being used in a report from Roche on testing of a new strategy to treat Parkinson鈥檚 disease.

The methods and tools from 丁香园AV may also help researchers develop a better understanding of other neurodegenerative diseases linked to GCase such as Dementia with Lewy Bodies (DLB) and Gaucher's Disease, a rare disease that usually manifests in childhood.

This work was sponsored by the Michael J. Fox Foundation for Parkinson鈥檚 Research and the Canadian Glycomics Network. The multinational Roche also collaborated on aspects of this work.