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Editing aberrant splice sites efficiently restores β-globin expression in β-thalassemia
The thalassemias are compelling targets for therapeutic genome editing in part because monoallelic correction of a subset of hematopoietic stem cells (HSCs) would be sufficient for enduring disease amelioration. A primary challenge is the development of efficient repair strategies that are effective...
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| Gepubliceerd in: | Blood |
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| Hoofdauteurs: | , , , , , , , , , , , |
| Formaat: | Artigo |
| Taal: | Inglês |
| Gepubliceerd in: |
American Society of Hematology
2019
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| Onderwerpen: | |
| Online toegang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6533605/ https://ncbi.nlm.nih.gov/pubmed/30704988 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2019-01-895094 |
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