Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells

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 sulfate and dermatan sulfate. Currently, the only approved t...

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Detalles Bibliográficos
Autores principales: Smith, Miles C., Belur, Lalitha R., Karlen, Andrea D., Erlanson, Olivia, Podetz-Pedersen, Kelly M., McKenzie, Jessica, Detellis, Jenn, Gagnidze, Khatuna, Parsons, Geoffrey, Robinson, Nicholas, Labarre, Shelby, Shah, Saumil, Furcich, Justin, Lund, Troy C., Tsai, Hsing-Chen, McIvor, R. Scott, Bonner, Melissa
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2022
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9808798/
https://www.ncbi.nlm.nih.gov/pubmed/36226412
http://dx.doi.org/10.1089/hum.2022.141

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