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AAV-shTAK1
- Development stage
- Preclinical
- Lead developer
- China Pharmaceutical University
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies
- Administration
- Intramuscular
01
Overview
AAV-shTAK1 is an adeno-associated virus (AAV) vector-based gene therapy designed to silence the expression of Transforming growth factor-beta-activated kinase 1 (TAK1) via short hairpin RNA (shRNA) interference. TAK1 is a key regulator of fibroblast and myoblast proliferation and differentiation, and its overactivation is associated with muscle wasting and fibrosis in Duchenne muscular dystrophy (DMD). By inhibiting TAK1, AAV-shTAK1 aims to prevent the transdifferentiation of myoblasts into fibroblasts, thereby reducing fibrosis and promoting muscle fiber regeneration. Research in mdx mouse models has demonstrated that TAK1 inhibition improves muscle quality and contractile function.
- Other names
- adeno-associated virus-short hairpin TAK1
02
Targets
TAK1 (Mitogen-activated protein kinase kinase kinase 7)Sialoglycan (Cell-surface sialoglycan)
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