Applications of the PMO platform to genetic diseases
نویسندگان
چکیده
منابع مشابه
Applications of the PMO platform to genetic diseases
Genetic diseases are caused by a variety of mutations some of which lead to aberrant splicing of premRNA and prevent proper mRNA translation of essential proteins or lead to translation of undesirable proteins. Such mRNA defects can be frequently repaired by appropriate targeting of oligonucleotides to modify splicing pathways and restore correct translation of desirable proteins. Hutchinson-Gi...
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ژورنال
عنوان ژورنال: Orphanet Journal of Rare Diseases
سال: 2015
ISSN: 1750-1172
DOI: 10.1186/1750-1172-10-s2-o34