Innovative Gene Therapy for
Parkinson's Disease
Parkinson's disease is a complex multifactorial condition, and the development of a successful therapy requires not only thoroughly considering the full spectrum of biological disturbances (factors) associated with the disease but also systematically ranking them by relative significance with a mandatory robust assessment of the scientific and experimental evidence pertaining to each.
Our understanding of the pathogenesis of Parkinson’s disease has been significantly refined in recent years. Drug research and development indicate that the predominant physiological disturbances are linked to the accumulation of pathological protein aggregates within neurons—primarily α-synuclein (Lewy bodies), but also ferritin aggregates and metal-binding proteins—which ultimately leads to neuronal loss, disruption of neurotransmitter balance, and the emergence of characteristic symptoms.