Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease

Fabry disease is an X-linked lysosomal storage disorder caused by loss of alpha-galactosidase A (α-Gal A) activity and is characterized by progressive accumulation of glycosphingolipids in multiple cells and tissues. FLT190, an investigational gene therapy, is currently being evaluated in a Phase 1/...

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Detalles Bibliográficos
Autores principales: Jeyakumar, Jey M., Kia, Azadeh, Tam, Lawrence C. S., McIntosh, Jenny, Spiewak, Justyna, Mills, Kevin, Heywood, Wendy, Chisari, Elisa, Castaldo, Noemi, Verhoef, Daniël, Hosseini, Paniz, Kalcheva, Petya, Cocita, Clement, Miranda, Carlos J., Canavese, Miriam, Khinder, Jaminder, Rosales, Cecilia, Hughes, Derralynn, Sheridan, Rose, Corbau, Romuald, Nathwani, Amit
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10284695/
https://www.ncbi.nlm.nih.gov/pubmed/36631545
http://dx.doi.org/10.1038/s41434-022-00381-y

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