Drug intelligence / Profile preview

alpha-synuclein A53T AAV

Development stage
Preclinical
Lead developer
GeneDetect
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intracerebral
01

Overview

Alpha-synuclein A53T AAV is an adeno-associated viral (AAV) vector used primarily in preclinical research to model Parkinson's disease. It delivers the human SNCA gene carrying the A53T missense mutation, which is associated with early-onset familial Parkinson's disease. Upon stereotaxic injection into specific brain regions like the substantia nigra pars compacta (SNpc), the vector induces the overexpression of mutant alpha-synuclein, leading to protein aggregation, dopaminergic neuronal loss, and mitochondrial dysfunction. This model is essential for investigating the molecular mechanisms of synucleinopathies and evaluating potential therapeutic interventions.

Other names
A53T alpha-synuclein adeno-associated virusA-53T alpha-synuclein adeno-associated virusA 53T alpha-synuclein adeno-associated virusAAV-A53T-alpha-synuclein expression vectorAAV-A-53T-alpha-synuclein expression vectorAAV-A 53T-alpha-synuclein expression vector
02

Targets

SNCA (Alpha-synuclein)

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