Key result
Intramuscular administration of AAV1-LPL(S447X) in LPL-deficient mice reduced plasma triglycerides from 99.0 to 1.8 mmol/L and increased HDL-C 6-fold, with effects lasting over 1 year.
Why the study?
Does AAV1-mediated gene transfer of hLPL(S447X) improve lipid profiles in a murine model of LPL deficiency?
Population
Adult murine model for LPL deficiency (LPL -/- mice)
Design
Preclinical
Follow-up
more than 1 year
Authors
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Supports muscle-directed gene therapy development for LPL deficiency; leaves open translation, safety, and efficacy in humans.
Does AAV1-mediated gene transfer of hLPL(S447X) improve lipid profiles in a murine model of LPL deficiency?
Absolute Event Rate: 1.8% vs 99%
AAV1-mediated gene transfer of LPL(S447X) into skeletal muscle results in long-term near-correction of dyslipidemia in a murine model of LPL deficiency.
Ross et al. (2004) studied Lipoprotein lipase (LPL) deficiency. AAV1-mediated expression of hLPL(S447X) (AAV1-LPL(S447X)) vs. Baseline/untreated state was evaluated on Plasma triglyceride (TG) levels. Intramuscular administration of AAV1-LPL(S447X) in LPL-deficient mice reduced plasma triglycerides from 99.0 to 1.8 mmol/L and increased HDL-C 6-fold, with effects lasting over 1 year.
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