Precision Medicine in Parkinson's Disease.

Area of Science:

• Neuroscience
• Genetics
• Pharmacology

Background:

• Parkinson's disease (PD) knowledge is growing, revealing genetic links and potential therapeutic targets.
• Genetic mutations in Glucosylceramidase beta 1 (GBA1) and Leucine-rich repeat kinase 2 (LRRK2) are significant risk factors for PD.
• These specific genetic forms are prevalent, making them attractive targets for drug development.

Purpose of the Study:

• To review current clinical trials and preclinical research focused on targeting genetic forms of Parkinson's disease.
• To examine therapeutic strategies aimed at the GBA1 and LRRK2 genes and their associated metabolic pathways.
• To explore the potential of these targeted therapies for sporadic PD cases.

Main Methods:

• Literature review of clinical trials and preclinical studies.
• Focus on studies investigating GBA1-associated PD and LRRK2-associated PD.
• Analysis of therapeutic mechanisms targeting these specific genes and pathways.

Main Results:

• Identification of ongoing and completed clinical trials for genetic PD.
• Evaluation of preclinical data supporting gene-specific and pathway-targeted approaches.
• Assessment of the potential for these therapies to impact sporadic PD.

Conclusions:

• Targeting GBA1 and LRRK2 represents a promising strategy for genetically defined Parkinson's disease.
• These gene-centric therapies hold potential for broader application in sporadic PD.
• Further research and clinical development are warranted for these targeted approaches.