The Designer Aminoglycoside NB84 Significantly Reduces Glycosaminoglycan Accumulation Associated with MPS I-H in the Idua-W392X Mouse

Suppression therapy utilizes compounds that suppress translation termination at in-frame premature termination codons (PTCs) to restore full-length, functional protein. This approach may provide a treatment for diseases caused by nonsense mutations such as mucopolysaccharidosis type I-Hurler (MPS I-...

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Huvudupphovsmän: Wang, Dan, Belakhov, Valery, Kandasamy, Jeyakumar, Baasov, Timor, Li, Su-Chen, Li, Yu-Teh, Bedwell, David M., Keeling, Kim M.
Materialtyp: Artigo
Språk:Inglês
Publicerad: 2011
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Article
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC3253910/
https://ncbi.nlm.nih.gov/pubmed/22056610
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2011.10.005
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