Lentiviral gene therapy reverts GPIX expression and phenotype in Bernard-Soulier syndrome type C.
Martinez-Navajas G, Ceron-Hernandez J, Simon I, Lupiañez P, Diaz-McLynn S, Perales S, Modlich U, Guerrero JA, Martin F, Sevivas T, Lozano ML, Rivera J, Ramos-Mejia V, Tersteeg C, Real PJ.
Martinez-Navajas G, et al. Among authors: tersteeg c.
Mol Ther Nucleic Acids. 2023 Jun 12;33:75-92. doi: 10.1016/j.omtn.2023.06.008. eCollection 2023 Sep 12.
Mol Ther Nucleic Acids. 2023.
PMID: 37416759
Free PMC article.