Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders
Vectors used in gene therapy for hemoglobin disorders carry globin in a reverse-orientation to prevent the loss of key regulatory elements by RNA splicing, but this limits their efficiency. Here, the authors develop a vector carrying β-globin in a forward orientation and show that it has improved ti...
| Main Authors: | , , , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Nature Publishing Group
2019-10-01
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| Series: | Nature Communications |
| Online Access: | https://doi.org/10.1038/s41467-019-12456-3 |