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In Vitro Modeling of GM1 Gangliosidosis Using Ipsc-Derived Cellular and Organoid CNS Models

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

Molecular Genetics and Metabolism（2023）

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