Key result
Intramuscular injection of an AAV7 vector expressing human apoE3 increased plasma apoE3 levels over 4 weeks, but concentrations remained just below the threshold needed to reduce hypercholesterolemia.
p-value: p=<0.001
Skeletal muscle can serve as a secretory platform for apoE3 transgene expression, but improved vectors are needed to achieve therapeutic plasma levels to reverse hypercholesterolemia.
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Demonstrates skeletal muscle as a feasible apoE3 secretory platform in animals; leaves open whether optimized vectors can reach therapeutic plasma levels.
Evans et al. (2008) studied Hypercholesterolemia / apoE deficiency. Intramuscular injection of nonviral DNA (plasmid) and AAV2/7 vectors expressing human apoE3 was evaluated on Local intramuscular expression of apoE3 and plasma apoE3 levels (p=<0.001). Intramuscular injection of an AAV7 vector expressing human apoE3 increased plasma apoE3 levels over 4 weeks, but concentrations remained just below the threshold needed to reduce hypercholesterolemia.
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