An engineered β-galactosidase with improved stability and cross-correction for the potential treatment of GM1 Gangliosidosis via AAV gene therapy.
- Resource Type
- Article
- Authors
- Reddy, Charu; Onguka, Ouma; Artz, Jacob; Schmitt, Christopher; Grossman, Adam; Santosh, Swetha; Akinkugbe, Oluwayomi; Wong, Victoria; Gebretsadik, Kahsay; Sedighi, Faezeh; Tang, Jinyan; Del Greco, Elizabeth; Lee, Grace S.; Kruse, Nikki; Chng, Chinping; Duan, Da; Hallows, William C.; Chen, Joseph C.; Akeefe, Hassibullah; Silverman, Adam P.
- Source
- Molecular Genetics & Metabolism. Feb2023, Vol. 138 Issue 2, pN.PAG-N.PAG. 1p.
- Subject
- *GENE therapy
*GALACTOSIDASES
*THERAPEUTICS
- Language
- ISSN
- 1096-7192