lipoprotein lipase adenovirus vector

A **lipoprotein lipase adenovirus vector** is an adenoviral gene therapy vector designed to deliver the human lipoprotein lipase (LPL) gene into patient tissues, typically liver or muscle, in order to restore or supplement LPL activity in individuals with LPL deficiency or related metabolic disorders. Mechanistically, this therapy uses a replication-defective adenovirus as a delivery vehicle to introduce an expression cassette for the human LPL gene (often the gain-of-function S447X variant) into host cells, thereby enabling endogenous production of functional LPL enzyme. This restores lipolytic activity and improves lipid clearance, reducing plasma triglycerides and ameliorating symptoms of severe hypertriglyceridemia. Adenovirus-mediated LPL delivery results in transient expression, since adenoviral vectors do not integrate into the host genome, and is mainly used as a preclinical or experimental approach. Adenovirus-based LPL gene therapy has demonstrated efficacy in animal studies and transient benefit in early clinical studies, including liver- and muscle-targeted approaches, but is typically limited by transient expression and immunogenicity[3][6].

Other names

lipoprotein lipase adenoviral vectorAd-LPLadenovirus-mediated LPL gene therapy