AAV-Syn-hTDP43-Php.Eb

AAV-Syn-hTDP43-Php.Eb is an experimental gene therapy vector comprising an adeno-associated virus (AAV) capsid of the engineered PHP.eB serotype, designed for efficient central nervous system (CNS) delivery, carrying a genetic payload encoding the human TAR DNA-binding protein 43 (hTDP-43) under the control of the human synapsin (Syn) promoter. The Syn promoter restricts transgene expression predominantly to neurons. TDP-43 is a DNA/RNA-binding protein implicated in several neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD); this vector allows for overexpression or modeling of TDP-43 proteinopathies in animal models. This tool is primarily used in research to model neurodegeneration and evaluate potential therapies targeting TDP-43 dysfunction. The AAV.PHP.eB capsid is notable for its superior ability to cross the blood-brain barrier and achieve widespread neuronal transduction in rodents, though its efficiency is highly species- and strain-dependent[2].