Olivia Erlanson
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View article: Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice
Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice Open
Mucopolysaccharidosis type II (MPS II) is an X-linked recessive lysosomal disease caused by iduronate-2-sulfatase (IDS) deficiency, leading to accumulation of glycosaminoglycans (GAGs) and the emergence of progressive disease. Enzyme repla…
View article: Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of <i>Ex Vivo</i> Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells
Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of <i>Ex Vivo</i> Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells Open
Mucopolysaccharidosis type II (MPS II, Hunter syndrome) is an X-linked recessive lysosomal disease caused by deficiency of iduronate-2-sulfatase (IDS). The absence of IDS results in the accumulation of the glycosaminoglycans (GAGs) heparan…