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An Engineered Β-Galactosidase with Improved Stability and Cross-Correction for the Potential Treatment of GM1 Gangliosidosis Via AAV Gene Therapy

Charu Reddy,Ouma Onguka,Jacob Artz,Christopher Schmitt, Adam Grossman,Swetha Santosh,Oluwayomi Akinkugbe,Victoria Wong,Kahsay Gebretsadik,Faezeh Sedighi, Jinyan Tang,Elizabeth Del Greco,Grace S. Lee,Nikki Kruse,Chinping Chng,Da Duan,William C. Hallows,Joseph C. Chen,Hassibullah Akeefe,Adam P. Silverman,Gjalt Huisman

MOLECULAR GENETICS AND METABOLISM（2023）

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Key words
Enzyme Replacement Therapy,Lysosomal Storage Disorders
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