Iduronate-2-sulfatase transport vehicle rescues behavioral and skeletal phenotypes in a mouse model of Hunter syndrome

Mucopolysaccharidosis type II (MPS II) is a lysosomal storage disorder caused by deficiency of the iduronate-2-sulfatase (IDS) enzyme, resulting in cellular accumulation of glycosaminoglycans (GAGs) throughout the body. Treatment of MPS II remains a considerable challenge as current enzyme replaceme...

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Detalles Bibliográficos
Autores principales: Arguello, Annie, Meisner, René, Thomsen, Elliot R., Nguyen, Hoang N., Ravi, Ritesh, Simms, Jeffrey, Lo, Iris, Speckart, Jessica, Holtzman, Julia, Gill, Thomas M., Chan, Darren, Cheng, Yuhsiang, Chiu, Chi-Lu, Dugas, Jason C., Fang, Meng, Lopez, Isabel A., Solanoy, Hilda, Tsogtbaatar, Buyankhishig, Zhu, Yuda, Bhalla, Akhil, Henne, Kirk R., Henry, Anastasia G., Delucchi, Anthony, Costanzo, Simona, Harris, Jeffrey M., Diaz, Dolores, Scearce-Levie, Kimberly, Sanchez, Pascal E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2021
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8525587/
https://www.ncbi.nlm.nih.gov/pubmed/34622797
http://dx.doi.org/10.1172/jci.insight.145445

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8525587/
https://www.ncbi.nlm.nih.gov/pubmed/34622797
http://dx.doi.org/10.1172/jci.insight.145445

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