An engineered β-galactosidase with improved stability and cross-correction for the potential treatment of GM1 Gangliosidosis via AAV gene therapy

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

doi:10.1016/j.ymgme.2022.107285

Molecular Genetics and Metabolism

2023

DOI: 10.1016/j.ymgme.2022.107285

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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 Reddy¹,

Ouma Onguka²,

Jacob H. Artz³

et al.

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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

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