Enzyme replacement therapy started at birth improves outcome in difficult-to-treat organs in mucopolysaccharidosis I mice
G Baldo, FQ Mayer, BZ Martinelli… - Molecular genetics and …, 2013 - Elsevier
Since we previously observed that in patients with mucopolysaccharidosis (MPS) the
storage of undegraded glycosaminoglycans (GAG) occurs from birth, in the present study we
aimed to compare normal, untreated MPS I mice (knockout for alpha-l-iduronidase-IDUA),
and MPS I mice treated with enzyme replacement therapy (ERT, Laronidase, 1.2 mg/kg
every 2weeks) started from birth (ERT-neo) or from 2months of age (ERT-ad). All mice were
sacrificed at 6months. Both treatments were equally effective in normalizing GAG levels in …
storage of undegraded glycosaminoglycans (GAG) occurs from birth, in the present study we
aimed to compare normal, untreated MPS I mice (knockout for alpha-l-iduronidase-IDUA),
and MPS I mice treated with enzyme replacement therapy (ERT, Laronidase, 1.2 mg/kg
every 2weeks) started from birth (ERT-neo) or from 2months of age (ERT-ad). All mice were
sacrificed at 6months. Both treatments were equally effective in normalizing GAG levels in …