Drug intelligence / Profile preview

scAAV9-CB-GFP

Development stage
Preclinical
Lead developer
Nationwide Children's Hospital
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intrathecal, Intracisternal, Intracerebroventricular
01

Overview

scAAV9-CB-GFP is a gene therapy research vector consisting of a self-complementary adeno-associated virus serotype 9 (scAAV9) capsid carrying an expression cassette with the chicken β-actin (CB) promoter driving enhanced green fluorescent protein (GFP) expression. It is used as a reporter construct in preclinical studies to map biodistribution and transduction efficiency following intrathecal or intracisternal administration, particularly in nonhuman primate models. The vector enables robust transgene expression primarily in the spinal cord, dorsal root ganglia, liver, striated muscle cells, cardiomyocytes, and some neurons—especially lower motor neurons and sensory neurons—while showing limited neuronal transduction in many brain regions. The GFP reporter allows for detailed mapping of gene delivery but does not have therapeutic intent; it serves as a surrogate for evaluating AAV-mediated gene transfer platforms that could later be adapted to deliver clinically relevant genes for neurological or systemic diseases[1][2][3].

Other names
self-complementary adeno-associated virus 9-chicken β-actin promoter-green fluorescent proteinscAAV9-CB-eGFPscAAV-9-CB-eGFPscAAV 9-CB-eGFP
02

Targets

Cell-surface glycans bearing terminal galactose

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