Ontology highlight
ABSTRACT:
SUBMITTER: Sorrentino NC
PROVIDER: S-EPMC6881609 | biostudies-literature | 2019 Dec
REPOSITORIES: biostudies-literature
Sorrentino Nicolina Cristina NC Cacace Vincenzo V De Risi Maria M Maffia Veronica V Strollo Sandra S Tedesco Novella N Nusco Edoardo E Romagnoli Noemi N Ventrella Domenico D Huang Yan Y Liu Nan N Kalled Susan L SL Choi Vivian W VW De Leonibus Elvira E Fraldi Alessandro A
Molecular therapy. Methods & clinical development 20191028
Mucopolysaccharidosis type IIIA (MPS-IIIA) is a lysosomal storage disorder (LSD) caused by inherited defect of sulfamidase, a lysosomal sulfatase. MPS-IIIA is one of the most common and severe forms of LSDs with CNS involvement. Presently there is no cure. Here we have developed a new gene delivery approach for the treatment of MPS-IIIA based on the use of a modified version of sulfamidase expression cassette. This cassette encodes both a chimeric sulfamidase containing an alternative signal pep ...[more]