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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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Detalles Bibliográficos
Publicado en:Diseases
Autor Principal: Keeling, Kim M.
Formato: Artigo
Idioma:Inglês
Publicado: MDPI 2016
Assuntos:
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC5370586/
https://ncbi.nlm.nih.gov/pubmed/28367323
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/diseases4040032
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