Biochemical Correction of GM2 Ganglioside Accumulation in AB-Variant GM2 Gangliosidosis
Deschenes, Natalie M, Cheng, Camilyn, Ryckman, Alex E, Quinville, Brianna M, Khanal, Prem, Mitchell, Melissa, Chen, Zhilin, Sangrar, Waheed, Gray, Steven J, Walia, Jagdeep S
Published in International journal of molecular sciences (24.05.2023)
Published in International journal of molecular sciences (24.05.2023)
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Journal Article
Efficacy of Adeno-Associated Virus Serotype 9-Mediated Gene Therapy for AB-Variant GM2 Gangliosidosis
Vyas, Meera, Deschenes, Natalie M, Osmon, Karlaina J. L, Chen, Zhilin, Ahmad, Imtiaz, Kot, Shalini, Thompson, Patrick, Richmond, Chris, Gray, Steven J, Walia, Jagdeep S
Published in International journal of molecular sciences (01.10.2023)
Published in International journal of molecular sciences (01.10.2023)
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Journal Article
Characterization of a phenotypically severe animal model for human AB-Variant GM2 gangliosidosis
Deschenes, Natalie M, Cheng, Camilyn, Khanal, Prem, Quinville, Brianna M, Ryckman, Alex E, Mitchell, Melissa, Pshezhetsky, Alexey V, Walia, Jagdeep S
Published in Frontiers in molecular neuroscience (30.11.2023)
Published in Frontiers in molecular neuroscience (30.11.2023)
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Intrathecal delivery of a bicistronic AAV9 vector expressing β-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study
Ryckman, Alex E., Deschenes, Natalie M., Quinville, Brianna M., Osmon, Karlaina J.L., Mitchell, Melissa, Chen, Zhilin, Gray, Steven J., Walia, Jagdeep S.
Published in Molecular therapy. Methods & clinical development (14.03.2024)
Published in Molecular therapy. Methods & clinical development (14.03.2024)
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