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AAV-hsyn-shMAF1
- Development stage
- Preclinical
- Lead developer
- Shanghai East Hospital
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies
- Administration
- Parenteral
01
Overview
AAV-hsyn-shMAF1 is an adeno-associated virus (AAV) vector-based gene therapy candidate designed to knock down the expression of MAF1 (Repressor of RNA polymerase III transcription MAF1 homolog) in neurons. It utilizes the human synapsin (hSyn) promoter to drive the expression of a short hairpin RNA (shRNA) targeting MAF1. MAF1 is a transcriptional regulator that is upregulated in neurological conditions such as acute ischemic stroke (AIS) and post-stroke cognitive impairment (PSCI). By knocking down MAF1, AAV-hsyn-shMAF1 inhibits NLRP3-mediated pyroptosis and enhances CREB-mediated neural repair, thereby attenuating ischemic cerebral injury and promoting functional recovery. The therapy is currently in preclinical development for the treatment of acute ischemic stroke, post-stroke cognitive impairment, and Alzheimer's disease.
- Other names
- AAV-hSyn-shMAF1AAV-hSyn-shMAF-1AAV-hSyn-shMAF 1AAV-syn-shMAF1AAV-syn-shMAF-1AAV-syn-shMAF 1
02
Targets
MAF1 (Repressor of RNA polymerase III transcription MAF1 homolog)
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