Stem cells used to identify potential treatments for Parkinson’s disease – Drug Target Review

Scientists have found two active substances that allowed cells to reactivate the production of a protein that prevents the onset of Parkinson’s disease.

Brain with Parkinson's

An international team of scientists has revealed the cause for certain genetic forms of Parkinson’s disease where PARK7 is mutated and has identified potential pharmacological treatments.

The team was led by Professor Rejko Krüger, of the Luxembourg Centre for Systems Biomedicine (LCSB) of the University of Luxembourg. The researchers experimented on patient-based cell cultures in the laboratory to discover their possible treatment. 

Previous research has shown that a protein called DJ-1 plays a crucial role in keeping nerve cells functioning. If the body is unable to produce ample amounts of DJ-1, important nerve cells die, resulting in the onset of neuro-degenerative diseases such as Parkinson’s. The production of important proteins like DJ-1 can be disrupted or halted permanently if the genetic blueprints or the production processes they encode are defective.

Krüger’s team succeeded in identifying for the first time the importance of an error in the production process known as ‘splicing’ in the development of a certain form of Parkinson’s disease.

“In [these Parkinson’s] patients, an essential tool for the assembly of the protein DJ-1 fails to dock properly,” Krüger explained. “In scientific terms, we call that exon skipping. As a result of this defect, the protein does not get built at all.”

The researchers say their findings