- In vitro restoration of functional SMN protein in human trophoblast cells affected by spinal muscular atrophy by small fragment homologous replacement. Sangiuolo, F., Filareto, A., Spitalieri, P., Scaldaferri, M.L., Mango, R., Bruscia, E., Citro, G., Brunetti, E., De Felici, M., Novelli, G. Hum. Gene Ther. (2005)