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Updated: May 14, 2025

Author Spotlight: Optimizing Digital Droplet PCR Method for Accurate Adeno-Associated Viral Genome Quantification
Published on: October 11, 2024
AAV gene therapy for mucopolysaccharidoses
Shaukat Khan1, Yasuhiko Ago1, Shunji Tomatsu2
1Nemours Children's Health, Wilmington, DE 19803, USA.
Abstract:
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.
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