Sabyasachi Das, Fatlum Rruga, Annita Montepeloso, Agnese Dimartino, Silvia Spadini, Guillaume Corre, Janki Patel, Eleonora Cavalca, Francesca Ferro, Alessandra Gatti, Rita Milazzo, Anne Galy, Letterio S Politi, Gian Paolo Rizzardi, Giuliana Vallanti, Valentina Poletti, Alessandra Biffi
Mucopolysaccharidosis type II (MPS II), or Hunter syndrome, is a rare X-linked recessive lysosomal storage disorder due to a mutation in the lysosomal enzyme iduronate-2-sulfatase (IDS) gene. IDS-deficiency leads to a progressive, multisystem accumulation of glycosaminoglycans (GAGs) and results in central nervous system (CNS) manifestations in the severe form. We developed up to clinical readiness a new Hematopoietic Stem Cell (HSC) gene therapy approach for MPS II that benefits from a novel highly effective transduction protocol...
February 2, 2024: Molecular Therapy