Drug intelligence / Profile preview

AAV-TAK1

Development stage
Preclinical
Lead developer
China Pharmaceutical University
Modality
Gene Therapies
Administration
Intramuscular
01

Overview

AAV-TAK1 is an adeno-associated virus (AAV) vector designed to overexpress Transforming growth factor-β-activated kinase 1 (TAK1), a protein encoded by the MAP3K7 gene. In the context of Duchenne muscular dystrophy (DMD) research, AAV-TAK1 is used as a tool to investigate the role of TAK1 activation in muscle pathology. Studies have shown that AAV-TAK1-mediated overexpression in mdx mice exacerbates muscle wasting and fibrosis by promoting the transdifferentiation of myoblasts into fibroblasts and inhibiting myoblast differentiation. This construct is primarily used in preclinical settings to validate TAK1 as a pathological driver and a potential therapeutic target, contrasting with TAK1 inhibitors (such as AAV-shTAK1 or NG25) which are being investigated for their ability to improve muscle quality and function in dystrophic skeletal muscle.

Other names
Adeno-associated virus-TAK1AAV-MAP3K7AAV-MAP-3K7AAV-MAP 3K7
02

Targets

IKK complexMKK3/6 (Dual specificity mitogen-activated protein kinase kinase 3 and 6)TAB2MKK4/7 (Mitogen-activated protein kinase kinase 4 and 7)

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