Codon-Optimization Enhances the Expression/Secretion of rSGSH, and Functional Benefits to Treat MPS IIIA in Mice via Systemic scAAV9-hSGSHop Gene Delivery

Tierra A. Bobo,Hyouk-Bum Kwon, Laura Montes, Christopher Tofade,Preston Samowitz,Haiyan Fu

MOLECULAR THERAPY(2023)

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