AAV Gene Therapy for MPS1-associated Corneal Blindness

Although cord blood transplantation has significantly extended the lifespan of mucopolysaccharidosis type 1 (MPS1) patients, over 95% manifest cornea clouding with about 50% progressing to blindness. As corneal transplants are met with high rejection rates in MPS1 children, there remains no treatmen...

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Bibliografische gegevens
Gepubliceerd in:Sci Rep
Hoofdauteurs: Vance, Melisa, Llanga, Telmo, Bennett, Will, Woodard, Kenton, Murlidharan, Giridhar, Chungfat, Neil, Asokan, Aravind, Gilger, Brian, Kurtzberg, Joanne, Samulski, R. Jude, Hirsch, Matthew L.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Nature Publishing Group 2016
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Article
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4761992/
https://ncbi.nlm.nih.gov/pubmed/26899286
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/srep22131
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