Engineered U7 Small Nuclear RNA Restores Correct β-Globin Pre-mRNA Splicing in Mouse β<sup>iVS2-654</sup>-Thalassemic Erythroid Progenitor Cells
Restoration of correct splicing of βIVS2-654-globin pre-mRNA was previously accomplished in erythroid cells from β-thalassemia/HbE patients by an engineered U7 small nuclear RNA (snRNA) that carried a sequence targeted to the cryptic branch point and an exonic splicing enhancer, U7.BP+623 snRNA. In...
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| Main Authors: | , , , , |
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| Format: | Article |
| Published: |
2022
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| Subjects: | |
| Online Access: | https://repository.li.mahidol.ac.th/handle/123456789/76199 |
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| Institution: | Mahidol University |