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Target intelligence / Profile preview
Alpha-synuclein Aggregates (α-syn Aggregates)
- Target
- α-syn Aggregates
- Molecular classification
- Protein Aggregate, Intracellular Inclusion
01
Overview
Alpha-synuclein aggregates are pathological accumulations of the alpha-synuclein protein, playing a central role in the development of Parkinson's disease and other synucleinopathies. These aggregates, including oligomers, protofibrils, and fibrils, disrupt cellular function and contribute to neurodegeneration. Targeting these aggregates is a major therapeutic strategy for these diseases.
- Other names
- Aggregated alpha-synucleinLewy bodiesAlpha-synuclein fibrilsAlpha-synuclein oligomersα-synuclein Aggregates
02
Mechanism of action
Targeting alpha-synuclein aggregates aims to reduce aggregate formation, promote aggregate clearance, or mitigate downstream toxicity. Strategies under investigation include small molecule inhibitors, antibodies, and gene therapies.
03
Biological functions
Disruption of synaptic vesicle traffickingImpairment of neurotransmitter releaseProteostasis impairmentMitochondrial dysfunctionAxonal transport disruptionNeurotoxicity
04
Disease associations
Parkinson's diseaseDementia with Lewy bodiesSynucleinopathies
05
Safety considerations
Potential for off-target effectsImmunogenicity of antibody-based therapiesDelivery challenges for brain-penetrant therapeuticsUnintended consequences of disrupting normal alpha-synuclein function
06
Biomarkers
Alpha-synuclein levels in cerebrospinal fluid (CSF)Alpha-synuclein levels in bloodAlpha-synuclein PET imaging
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