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AAV9-Cdk5-shRNA
- Development stage
- Preclinical
- Lead developer
- Seoul National University Hospital
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies
- Administration
- Intrathecal
01
Overview
AAV9-Cdk5-shRNA is an investigational gene therapy consisting of an adeno-associated virus serotype 9 (AAV9) vector delivering a short hairpin RNA (shRNA) designed to silence cyclin-dependent kinase 5 (Cdk5). Developed by researchers at Seoul National University and Seoul National University Hospital, the therapy is designed to treat amyotrophic lateral sclerosis (ALS). By inhibiting Cdk5, the therapy aims to suppress tau hyperphosphorylation, neuroinflammation, and apoptosis, thereby preventing motor neuron degeneration and extending survival.
02
Targets
CDK5 (Cyclin-dependent kinase 5)
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