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Nonsense Suppression as an Approach to Treat Lysosomal Storage Diseases
In-frame premature termination codons (PTCs) (also referred to as nonsense mutations) comprise ~10% of all disease-associated gene lesions. PTCs reduce gene expression in two ways. First, PTCs prematurely terminate translation of an mRNA, leading to the production of a truncated polypeptide that oft...
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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MDPI
2016
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5370586/ https://www.ncbi.nlm.nih.gov/pubmed/28367323 http://dx.doi.org/10.3390/diseases4040032 |