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Hammerhead ribozymes selectively suppress mutant type I collagen mRNA in osteogenesis imperfecta fibroblasts

Ribozymes are a promising agent for the gene therapy of dominant negative genetic disorders by allele-specific mRNA suppression. To test allele-specific mRNA suppression in cells, we used fibroblasts from a patient with osteogenesis imperfecta (OI). These cells contain a mutation in one α1(I) collag...

詳細記述

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書誌詳細
出版年:Nucleic Acids Res
主要な著者: Dawson, Paul A., Marini, Joan C.
フォーマット: Artigo
言語:Inglês
出版事項: Oxford University Press 2000
主題:
オンライン・アクセス:https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC110781/
https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/11024182/
https://ncbi.nlm.nih.govhttps://doi.org/10.1093/nar/28.20.4013
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