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AAV-GLT-1-shRNA
- Development stage
- Preclinical
- Lead developer
- Soochow University
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Parenteral
01
Overview
AAV-GLT-1-shRNA is a gene therapy tool consisting of an adeno-associated virus (AAV) vector that delivers short hairpin RNA (shRNA) targeting the glutamate transporter-1 (GLT-1), also known as excitatory amino acid transporter 2 (EAAT2) or SLC1A2. Primarily utilized in preclinical research, this agent is designed to selectively down-regulate GLT-1 expression in specific brain regions, such as the striatum. The resulting deficiency in astrocytic glutamate uptake leads to glutamate accumulation, excitotoxicity, and subsequent dopaminergic neurodegeneration. This mechanism is employed to create experimental models of Parkinson's disease that mimic the astrocytic dysfunction and excitatory amino acid toxicity observed in the human condition.
- Other names
- AAV-shGLT-1AAV-shGLT1AAV-shGLT 1AAV-GLT1-shRNAAAV-GLT-1-shRNAAAV-GLT 1-shRNA
02
Targets
SLC1A2 (Glutamate Transporter 1)
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