Haematopoietic stem cell gene therapy with IL‐1Ra rescues cognitive loss in mucopolysaccharidosis IIIA

por Parker, Helen, Ellison, Stuart M, Holley, Rebecca J, O'Leary, Claire, Liao, Aiyin, Asadi, Jalal, Glover, Emily, Ghosh, Arunabha, Jones, Simon, Wilkinson, Fiona L, Brough, David, Pinteaux, Emmanuel, Boutin, Hervé, Bigger, Brian W
Publicado 2020

Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA

por Ellison, Stuart M., Liao, Aiyin, Wood, Shaun, Taylor, Jessica, Youshani, Amir Saam, Rowlston, Sam, Parker, Helen, Armant, Myriam, Biffi, Alessandra, Chan, Lucas, Farzaneh, Farzin, Wynn, Rob, Jones, Simon A., Heal, Paul, Gaspar, H. Bobby, Bigger, Brian W.
Publicado 2019