Next-generation sequencing and recombinant expression characterized aberrant splicing mechanisms and provided correction strategies in factor VII deficiency.
Haematologica
Ferraresi, Paolo P; Balestra, Dario D; Guittard, Caroline C; Buthiau, Delphine D; Pan-Petesh, Brigitte B; Maestri, Iva I; Farah, Roula R; Pinotti, Mirko M; Giansily-Blaizot, Muriel M