Drug intelligence / Profile preview

AAV8-TGF-β1

Development stage: Preclinical
Modality: Gene Therapies
Administration: Intravenous

Overview

AAV8-TGF-β1 is an adeno-associated virus serotype 8 (AAV8) viral vector designed to deliver and overexpress transforming growth factor-beta 1 (TGF-β1). In preclinical research, it is widely utilized as an in vivo model to induce chronic TGF-β1 signaling, leading to hepatocellular injury, loss of metabolic zonation, liver sinusoidal endothelial cell (LSEC) capillarization, and hepatic stellate cell activation, thereby establishing a profibrogenic hepatic microenvironment. Additionally, AAV8-mediated delivery of TGF-β1 has been investigated in preclinical models of retinal degeneration, where it was shown to promote cone photoreceptor survival and improve visual function by modulating microglial activation and metabolic programming.

Other names: AAV8-TGF-betaAAV-8-TGF-betaAAV 8-TGF-betaAAV8-TGF-beta1AAV-8-TGF-beta1AAV 8-TGF-beta1AAV8-TGF-βadeno-associated virus serotype 8-transforming growth factor-beta 1

Targets

TGFBR1TGFBR2 (TGF-β receptor type 2)

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