VIRON Biosciences

What is Parkinson's Disease?

Parkinson’s disease is a chronic neurodegenerative disorder that involves both motor and non-motor symptoms. Symptoms include tremors, stiffness, slurred speech, cognitive decline, but symptoms vary per person (Li et al., 2022). In recent years epigenetic mechanisms have been linked to this disease (Song et al., 2023). This disease has an effect on motor skills because dopaminergic neurons are being degraded (Jowaed et al., 2010). Symptoms progressively get worse, but many treatments are being offered to mitigate the negative effects of this disease.

FIGURE 3: (https://www.porterhousemedical.com/news/parkinsons-disease-a-hopeful-future/)

Genetic Influence

There are many well known genetic causes linked to Parkinson’s. Epigenetic mechanisms are still being researched. Parkinson’s is associated with mutations in LRRK2, PRKN, and SNCA (Jowaed et al., 2010). 

Mutations in leucine-rich repeat kinase 2 (LRRK2) cause neurodegeneration from improper cell signaling. Parkin (PRKN) helps control the function of the mitochondria (Rui et al., 2018), mutations in this gene lead to poor mitochondrial health (Song et al., 2023). Alpha-synuclein (SNCA) mutations result in excess alpha-synuclein which clump and damage neurons (Rui et al., 2018).

FIGURE 4: (https://wi.mit.edu/news/identifying-genetic-mutation-behind-sporadic-parkinson-s-disease)

Treatments

CARBIDOPA-LEVODOPA
            Provides sufficient dopamine to the brain (IV or inhaled)

DOPAMINE AGONISTS
            - Rotigotine   - Pramipexole

CATECHOL O-METHYLTRANSFERASE INHIBITORS (COMT) 
           (Inhibits dopamine breakdown)
            - Entacapone   - Tolcapone 

(MAYO Clinic, 2023)

Innovative Research

VIRON is currently researching gene therapies for early-onset Parkinson’s. These gene therapies focus on targeting SNCA, LRRK2, and PRKN mutations, in hopes of inducing proper protein function (Jowaed et al., 2010). 

For long term results CRISPR editing can be utilized to prevent protein buildup in neurons. This would be done by silencing genes responsible for producing these harmful protein clusters. 

Researchers are looking into the use of HDAC inhibitors and other epigenetic modulators to reduce inflammation, and prevent the degradation of dopaminergic neurons (Song et al., 2023).