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AAV-Ep+-IgG-GFP is a gene therapy research construct consisting of an adeno-associated virus (AAV) vector with a novel AAV-Ep+ capsid that delivers a gene encoding an immunoglobulin G (IgG) antibody targeting Green Fluorescent Protein (GFP). It is primarily utilized as an isotype-control in preclinical studies investigating AAV-delivered antibody therapies for neurodegenerative diseases, such as C9orf72-associated amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The AAV-Ep+ capsid is specifically designed for high-efficiency transduction of neurons and ependymal cells in the brain and spinal cord, enabling the secretion of the encoded antibody into the cerebrospinal fluid (CSF). In experimental settings, this construct serves to establish a baseline for the effects of the viral delivery system and the expression of a non-therapeutic antibody, allowing for the rigorous evaluation of target-specific candidates like AAV-Ep+IgG1-GA.
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