LAMP2A gene therapy reduces Parkinson's disease symptoms in primates

Increasing LAMP2A expression in nonhuman primates protected nigral dopaminergic neurons from α-synuclein neurodegeneration.

• LAMP2A is a receptor that regulates chaperone-mediated autophagy, crucial for clearing α-synuclein.
• Rodent studies indicate that enhancing LAMP2A can reverse neurodegeneration, while its deficiency can worsen pathology.
• Injections of Parkinson's disease patient-derived Lewy body extract induced α-synucleinopathy in the primate model.
• Increased LAMP2A expression delayed the formation and release of harmful α-synuclein species.
• A beneficial effect on motor behavior and cognitive functions was observed in the primate model.

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