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pro vyhledávání: '"Kassie S. Manning"'
Publikováno v:
ACS Chemical Biology. 12:2503-2509
Myotonic dystrophy type 1 (DM1) is a multisystemic disease caused by an expanded CTG repeat in the 3′ UTR of the dystrophia myotonica protein kinase (DMPK) gene. Short, DNA-based antisense oligonucleotides termed gapmers are a promising strategy to
Publikováno v:
ACS chemical biology. 12(10)
Myotonic dystrophy type 1 (DM1) is a multisystemic disease caused by an expanded CTG repeat in the 3′ UTR of the dystrophia myotonica protein kinase (DMPK) gene. Short, DNA-based antisense oligonucleotides termed gapmers are a promising strategy to
Autor:
Thomas A. Cooper, Kassie S. Manning
Publikováno v:
Nature reviews. Molecular cell biology. 18(2)
A goal of human genetics studies is to determine the mechanisms by which genetic variation produces phenotypic differences that affect human health. Efforts in this respect have previously focused on genetic variants that affect mRNA levels by alteri