Lentiviral gene therapy reverts GPIX expression and phenotype in Bernard-Soulier syndrome type C

Bernard-Soulier syndrome (BSS) is a rare congenital disease characterized by macrothrombocytopenia and frequent bleeding. It is caused by pathogenic variants in three genes (GP1BA, GP1BB, or GP9) that encode for the GPIbα, GPIbβ, and GPIX subunits of the GPIb-V-IX complex, the main platelet surface...

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Detalles Bibliográficos
Autores principales: Martinez-Navajas, Gonzalo, Ceron-Hernandez, Jorge, Simon, Iris, Lupiañez, Pablo, Diaz-McLynn, Sofia, Perales, Sonia, Modlich, Ute, Guerrero, Jose A., Martin, Francisco, Sevivas, Teresa, Lozano, Maria L., Rivera, Jose, Ramos-Mejia, Veronica, Tersteeg, Claudia, Real, Pedro J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10320622/
https://www.ncbi.nlm.nih.gov/pubmed/37416759
http://dx.doi.org/10.1016/j.omtn.2023.06.008

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