Intrathecal delivery of a bicistronic AAV9 vector expressing β-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study
Article in Molecular Therapy — Methods & Clinical Development (December 2023)
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Article in Molecular Therapy — Methods & Clinical Development (December 2023)
Book chapter (September 2023)
Article in Biomolecules (August 2023)