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