Neonatal gene therapy effectively prevents disease manifestations in a murine model of Mucopolysaccharidosis type I.

ABSTRACT: Mucopolysaccharidosis type I (MPS-I) is a rare pediatric disease caused by mutations in the α-L-iduronidase (IDUA) gene encoding for a lysosomal enzyme involved in glycosaminoglycan metabolism. While newborns with the severe Hurler variant are usually asymptomatic at birth, progressive disease manifestations emerge early in life. Since previous studies on lentiviral vector gene therapy (GT) in Hurler patients have demonstrated superior metabolic correction and early beneficial clinical effects, we investigated whether applying this GT approach during the neonatal period could be effective in preventing disease pathology before it becomes irreversible. Thus, newborn MPS-I mice were transplanted with affected bone marrow-derived progenitor cells transduced with an IDUA-encoding lentiv

SUBMITTER: De Ponti G 

PROVIDER: S-EPMC12357110 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature