AAV6 Serotype

Natural serotype with exceptionally strong skeletal muscle, cardiac muscle, and lung tropism. Clinically relevant for muscle and airway diseases.

Length: 1 bp

Tropism: Skeletal muscle, cardiac muscle, lung, liver (moderate)

Characteristics

Highest skeletal muscle transduction among natural serotypes. Strong cardiac muscle tropism. Efficient lung and airway epithelium transduction. Uses HSPG and sialic acid receptors. Moderate pre-existing immunity (~50% seropositive). Foundation for tyrosine-mutant engineering (Y445F, Y731F improvements). Good manufacturing properties.

Applications

Duchenne muscular dystrophy (clinical trials). Limb-girdle muscular dystrophies. Hemophilia (muscle-directed Factor VIII/IX expression). Cystic fibrosis lung gene therapy. Cardiac gene therapy. Most common serotype for muscle disease trials.

Literature References

  1. Rutledge et al. (1998). Infectious clones and vectors derived from adeno-associated virus (AAV) serotypes other than AAV type 2. J Virol - Rutledge 1998 AAV6