Gene Therapy to Restore Parkin Protein in Early-Onset Parkinson's Disease

Biallelic loss-of-function mutations in the PRKN gene are associated with early-onset Parkinson's disease.

• The PRKN gene encodes the Parkin protein, which plays a key role in the removal of damaged mitochondria.
• Mutations in the PRKN gene lead to a functional deficiency of Parkin, impairing mitochondrial degradation.
• Accumulation of damaged mitochondria due to lack of Parkin may result in increased oxidative stress.
• Oxidative stress is linked to neurodegeneration seen in early-onset Parkinson's disease.

Simplified