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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Veröffentlicht in:	Gene therapy 2023-06, Vol.30 (6), p.487-502
Hauptverfasser:	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
Format:	Artikel
Sprache:	eng
Schlagworte:	13/109 13/51 14/28 14/63 42 42/41 42/44 64/110 692/699 692/699/317 a-Galactosidase alpha-Galactosidase - genetics alpha-Galactosidase - metabolism Animal models Animals Biodistribution Biomedical and Life Sciences Biomedicine Cell Biology Cells, Cultured Electron microscopy Fabry Disease - genetics Fabry Disease - therapy Fabry's disease Fibroblasts Gene Expression Gene Therapy Genetic Therapy Genetic Vectors Genomes Globotriaosylceramide Glycosphingolipids Human Genetics Humans Inclusion bodies Kidneys Liver Liver - metabolism Mice mRNA Nanotechnology Signal transduction Vectors (Biology)
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