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Correction of Beta-Thalassemia IVS-II-654 Mutation in a Mouse Model Using Prime Editing
Prime editing was used to insert and correct various pathogenic mutations except for beta-thalassemia variants, which disrupt functional beta-globin and prevent hemoglobin assembly in erythrocytes. This study investigated the effect of gene correction using prime editor version 3 (PE3) in a mouse mo...
| Autores principales: | , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
MDPI
2022
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9180235/ https://www.ncbi.nlm.nih.gov/pubmed/35682629 http://dx.doi.org/10.3390/ijms23115948 |