AAV1 Serotype

Natural AAV serotype with strong skeletal and cardiac muscle tropism. One of the earliest discovered and most clinically relevant serotypes for muscle-directed gene therapy.

Length: 1 bp

Tropism: Skeletal muscle, cardiac muscle, CNS (moderate)

Characteristics

High skeletal muscle transduction efficiency. Strong cardiac muscle tropism. Moderate CNS penetration following direct injection. Heparan sulfate proteoglycan (HSPG) binding. Moderate pre-existing immunity in human populations (~40% seropositive). Uses sialic acid as cellular receptor.

Applications

Limb-girdle muscular dystrophy (clinical trials). Alpha-1 antitrypsin deficiency (Glybera - first approved AAV gene therapy in EU, now withdrawn). Lipoprotein lipase deficiency. Duchenne muscular dystrophy. Heart failure gene therapy.

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