AAV gene therapy for mucopolysaccharidoses.

Adeno-associated viral (AAV) vectors emerge as a promising treatment option for genetic diseases. They are recognized as the most effective in vivo gene therapy due to their safety, efficacy, and ability to provide long-term therapeutic transgene expression.1,2,3 Rossi et al.1 have demonstrated that AAV gene therapy is effective and safe for treating patients with mucopolysaccharidosis VI, paving the way for potential treatments for mucopolysaccharidoses.