Scientists elucidate cause for Parkinson's disease and identify potential treatments

[Farooqji]

In a seven-year research effort, an international team of scientists has clarified the cause for certain genetic forms of Parkinson's disease, and has identified potential pharmacological treatments.The interdisciplinary research team, led by Prof. Rejko Krüger, of the Luxembourg Centre for Systems Biomedicine (LCSB) of the University of Luxembourg, experimented on patient-based cell cultures in the laboratory. The new combination of active substances they identified will have to undergo clinical trials before they can be used to treat patients. The research team published its results today in the prestigious scientific journal Science Translational Medicine.

news-medical.net/amp/news/2...

[park_bear]

For patients with a mutation in the park7 gene:

stm.sciencemag.org/content/...

"PARK(7) preservation

Mutations in PARK7 lead to the development of early-onset Parkinson’s disease (PD), a neurodegenerative condition for which there are currently no effective treatments. Here, Boussaad et al. identified an exonic splicing mutation in PARK7 linked to PD and studied the effect of this mutation in patient-derived cellular models. The mutation resulted in impaired splicing, reduced production of DJ-1 protein, and consequent mitochondrial dysfunction. Rescuing the aberrant splicing with the kinetin analog RECTAS in combination with phenylbutyric acid rescued neuronal loss in patient-derived brain organoids. The results suggest that precision medicine targeting specific molecular signatures could be an effective strategy for PD and possibly other neurodegenerative diseases."

[lupacexi]

Thanks you for sharing