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Long-term nonsense suppression therapy moderates MPS I-H disease progression.
Gunn G, Dai Y, Du M, Belakhov V, Kandasamy J, Schoeb TR, Baasov T, Bedwell DM, Keeling KM. Gunn G, et al. Among authors: belakhov v. Mol Genet Metab. 2014 Mar;111(3):374-381. doi: 10.1016/j.ymgme.2013.12.007. Epub 2013 Dec 17. Mol Genet Metab. 2014. PMID: 24411223 Free PMC article.
We previously showed that 2-week treatment with the designer aminoglycoside NB84 restored enough alpha-L-iduronidase function via PTC suppression to reduce tissue GAG accumulation in the Idua(tm1Kmke) MPS I-H mouse model, which carries a PTC homologous to the human IDUA-W402X non …
We previously showed that 2-week treatment with the designer aminoglycoside NB84 restored enough alpha-L-iduronidase function via PTC suppre …