GM1 Gangliosidosis: Mechanisms and Management
Author:
Publisher
Informa UK Limited
Subject
Genetics (clinical),Genetics
Reference192 articles.
1. GM1 gangliosidosis and Morquio B disease: An update on genetic alterations and clinical findings
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3. MRI/MRS as a surrogate marker for clinical progression in GM1 gangliosidosis
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1. Insights into the Pathobiology of GM1 Gangliosidosis from Single-Nucleus Transcriptomic Analysis of CNS Cells in a Mouse Model;International Journal of Molecular Sciences;2024-09-08
2. Validation of high-sensitivity assays to quantitate cerebrospinal fluid and serum β-galactosidase activity in patients with GM1-gangliosidosis;Molecular Therapy - Methods & Clinical Development;2024-09
3. Generation of an infantile GM1 gangliosidosis induced pluripotent stem cell line (CHOCi005-A) for disease modeling and therapeutic testing;Stem Cell Research;2024-09
4. Base editing of the GLB1 gene is therapeutic in GM1 gangliosidosis patient-derived cells;Molecular Genetics and Metabolism;2024-09
5. Differential Tractography: A Biomarker for Neuronal Function in Neurodegenerative Disease;2024-08-26
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