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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 …
Poly-L-aspartic acid enhances and prolongs gentamicin-mediated suppression of the CFTR-G542X mutation in a cystic fibrosis mouse model.
Du M, Keeling KM, Fan L, Liu X, Bedwell DM.
Du M, et al. Among authors: bedwell dm.
J Biol Chem. 2009 Mar 13;284(11):6885-92. doi: 10.1074/jbc.M806728200. Epub 2009 Jan 9.
J Biol Chem. 2009.
PMID: 19136563
Free PMC article.
Because the use of gentamicin to suppress disease-causing nonsense mutations will require their long term administration, the ability of PAA to reduce toxicity and increase both the level and duration of readthrough has important implications for this promising therapeutic …
Because the use of gentamicin to suppress disease-causing nonsense mutations will require their long term administration, the ability …
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