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...
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| Veröffentlicht in: | Nucleic Acids Res |
|---|---|
| Hauptverfasser: | , |
| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
Oxford University Press
2000
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| Online-Zugang: | 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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