Results 131 to 140 of about 12,041 (191)

Mucopolysaccharidosis type II zebrafish model exhibits early impaired proteasomal-mediated degradation of the axon guidance receptor Dcc. [PDF]

open access: yesCell Death Dis
Manzoli R   +7 more
europepmc   +1 more source

Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice. [PDF]

open access: yesMol Ther Methods Clin Dev
Smith MC   +11 more
europepmc   +1 more source

An empowered, clinically viable hematopoietic stem cell gene therapy for the treatment of multisystemic mucopolysaccharidosis type II. [PDF]

open access: yesMol Ther
Das S   +16 more
europepmc   +1 more source

Modeling Lysosomal Storage Disorders in an Innovative Way: Establishment and Characterization of Stem Cell Lines from Human Exfoliated Deciduous Teeth of Mucopolysaccharidosis Type II Patients. [PDF]

open access: yesInt J Mol Sci
Carvalho S   +20 more
europepmc   +1 more source

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