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RNA repair restores hemoglobin expression in IVS2–654 thalassemic mice
Repair of β-globin pre-mRNA rendered defective by a thalassemia-causing splicing mutation, IVS2–654, in intron 2 of the human β-globin gene was accomplished in vivo in a mouse model of IVS2–654 thalassemia. This was effected by a systemically delivered splice-switching oligonucleotide (SSO), a morph...
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| Hlavní autoři: | , , , , , , , |
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| Médium: | Artigo |
| Jazyk: | Inglês |
| Vydáno: |
National Academy of Sciences
2009
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| Témata: | |
| On-line přístup: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2633555/ https://ncbi.nlm.nih.gov/pubmed/19164558 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0812436106 |
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