Repair of thalassemic human β-globin mRNA in mammalian cells by antisense oligonucleotides
In one form of β-thalassemia, a genetic blood disorder, a mutation in intron 2 of the β-globin gene (IVS2-654) causes aberrant splicing of β-globin pre-mRNA and, consequently, β-globin deficiency. Treatment of mammalian cells stably expressing the IVS2-654 human β-globin gene with antisense oligonuc...
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| 發表在: | Proc Natl Acad Sci U S A |
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| Principais autores: | , , , |
| 格式: | Artigo |
| 語言: | Inglês |
| 出版: |
National Academy of Sciences
1996
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| 主題: | |
| 在線閱讀: | https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC24007/ https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/8917506/ https://ncbi.nlm.nih.govhttps://doi.org/10.1073/pnas.93.23.12840 |
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