A pentasaccharide for monitoring pharmacodynamic response to gene therapy in GM1 gangliosidosis

BACKGROUND: GM1 gangliosidosis is a rare, fatal, neurodegenerative disease caused by mutations in the GLB1 gene and deficiency in β-galactosidase. Delay of symptom onset and increase in lifespan in a GM1 gangliosidosis cat model after adeno-associated viral (AAV) gene therapy treatment provide the b...

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Detalles Bibliográficos
Autores principales: Kell, Pamela, Sidhu, Rohini, Qian, Mingxing, Mishra, Sonali, Nicoli, Elena-Raluca, D'Souza, Precilla, Tifft, Cynthia J., Gross, Amanda L., Gray-Edwards, Heather L., Martin, Douglas R., Sena- Esteves, Miguel, Dietzen, Dennis J., Singh, Manmilan, Luo, Jingqin, Schaffer, Jean E., Ory, Daniel S., Jiang, Xuntian
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2023
Materias:
Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10277919/
https://www.ncbi.nlm.nih.gov/pubmed/37267847
http://dx.doi.org/10.1016/j.ebiom.2023.104627

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