Intrastromal Gene Therapy Prevents and Reverses Advanced Corneal Clouding in a Canine Model of Mucopolysaccharidosis I

Mucopolysaccharidosis type I (MPS I) is an autosomal recessive lysosomal storage disease characterized by severe phenotypes, including corneal clouding. MPS I is caused by mutations in alpha-l-iduronidase (IDUA), a ubiquitous enzyme that catalyzes the hydrolysis of glycosaminoglycans. Currently, no...

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Bibliografiska uppgifter
I publikationen:Mol Ther
Huvudupphovsmän: Miyadera, Keiko, Conatser, Laura, Llanga, Telmo A., Carlin, Kendall, O’Donnell, Patricia, Bagel, Jessica, Song, Liujiang, Kurtzberg, Joanne, Samulski, R. Jude, Gilger, Brian, Hirsch, Matthew L.
Materialtyp: Artigo
Språk:Inglês
Publicerad: American Society of Gene & Cell Therapy 2020
Ämnen:
Original Article
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC7264440/
https://ncbi.nlm.nih.gov/pubmed/32330426
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.04.004
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