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Efficient and specific repair of sickle β-globin RNA by trans-splicing ribozymes
Previously we demonstrated that a group I ribozyme can perform trans-splicing to repair sickle β-globin transcripts upon transfection of in vitro transcribed ribozyme into mammalian cells. Here, we sought to develop expression cassettes that would yield high levels of active ribozyme after gene tran...
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| Main Authors: | , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Copyright 2003 by RNA Society
2003
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC1370489/ https://ncbi.nlm.nih.gov/pubmed/13130139 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1261/rna.5450203 |
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