It appears to have been confirmed that the α-Synuclein gene is the one primarily linked to Parkinson’s disease, while the G209 (p.A53T) mutation of this gene causes a type of Parkinson’s disease characterized by early and severe onset of symptoms. It has now been reported (see HERE) that iPS cells have been produced from patients with the aforementioned p.A53T deficiency, allowing scientists to increase their understanding of the pathology of this disease. In turn, it has been shown that small molecules that target the α-Synuclein gene can reverse the degenerative phenotype under both basal and stress conditions. Furthermore, mutant neurons showed disrupted synaptic connectivity and widespread transcriptional alterations in genes involved in synaptic signaling, a number of which have been previously linked to mental disorders, raising intriguing implications for potentially converging disease mechanisms.

These iPS cells new applications undoubtedly open new possibilities for better understanding of this kind of Parkinson’s disease and for a hypothetical treatment, which has the ethical — as well as medical — advantage that these findings have been made using iPS cells. Furthermore, mutant neurons showed disrupted synaptic connectivity and widespread transcriptional alterations in genes involved in synaptic signaling, a number of which have been previously linked to mental disorders, raising intriguing implications for potentially converging disease mechanisms.