Ontology highlight
ABSTRACT:
SUBMITTER: Squeri G
PROVIDER: S-EPMC6612662 | biostudies-literature | 2019 Jul
REPOSITORIES: biostudies-literature
Squeri Giorgia G Passerini Laura L Ferro Francesca F Laudisa Cecilia C Tomasoni Daniela D Deodato Federica F Donati Maria Alice MA Gasperini Serena S Aiuti Alessandro A Bernardo Maria Ester ME Gentner Bernhard B Naldini Luigi L Annoni Andrea A Biffi Alessandra A Gregori Silvia S
Molecular therapy : the journal of the American Society of Gene Therapy 20190419 7
Mucopolysaccharidosis type I (MPS-I) is a severe genetic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) enzyme. Ex vivo hematopoietic stem cell (HSC) gene therapy is a promising therapeutic approach for MPS-I, as demonstrated by preclinical studies performed in naive MPS-I mice. However, after enzyme replacement therapy (ERT), several MPS-I patients develop anti-IDUA immunity that may jeopardize ex vivo gene therapy efficacy. Here we treat MPS-I mice with an artificial immuniza ...[more]