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Long-term nonsense suppression therapy moderates MPS I-H disease progression.
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 …
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