Therapeutic advances in musculoskeletal AAV targeting approaches
K Bulaklak, X Xiao - Current Opinion in Pharmacology, 2017 - Elsevier
Highlights•Recombinant AAV therapies are highly promising gene for musculoskeletal
disease.•Mutant capsids evade neutralization and sequestration to increase
benefit.•Regulatory elements improve rAAV muscle-specific expression.•Pharmacological
agents can improve rAAV therapeutic efficacy.•Improved preclinical models highlight the
advantages of rAAV vectors.The use of recombinant adeno-associated viruses (rAAVs) is
highly prevalent in musculoskeletal gene therapies due to their versatility, high transduction …
disease.•Mutant capsids evade neutralization and sequestration to increase
benefit.•Regulatory elements improve rAAV muscle-specific expression.•Pharmacological
agents can improve rAAV therapeutic efficacy.•Improved preclinical models highlight the
advantages of rAAV vectors.The use of recombinant adeno-associated viruses (rAAVs) is
highly prevalent in musculoskeletal gene therapies due to their versatility, high transduction …