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Restoration of correct β(IVS2-654)-globin mRNA splicing and HbA production by engineered U7 snRNA in β-thalassaemia/HbE erythroid cells
A cytosine to thymine mutation at nucleotide 654 of human β-globin intron 2 (β(IVS2-654)) is one of the most common mutations causing β-thalassaemia in Chinese and Southeast Asians. This mutation results in aberrant β-globin pre-mRNA splicing and prevents synthesis of β-globin protein. Splicing corr...
| Autores principales: | , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Nature Publishing Group UK
2019
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6529457/ https://www.ncbi.nlm.nih.gov/pubmed/31113996 http://dx.doi.org/10.1038/s41598-019-43964-3 |