Nonsense Suppression as an Approach to Treat Lysosomal Storage Diseases
نویسندگان
چکیده
منابع مشابه
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 often lacks normal function and/or is unstable. Second, PTCs trigger degradation of an mRNA by activ...
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ژورنال
عنوان ژورنال: Diseases
سال: 2016
ISSN: 2079-9721
DOI: 10.3390/diseases4040032