Sequence-specific modification of a β-thalassemia locus by small DNA fragments in human erythroid progenitor cells
Gene therapy has been proposed as a definitive cure for β-thalassemia. We applied a gene targeting approach, based on the introduction of small DNA fragments (SDF) into erythroid progenitor cells, to specifically modify the β-globin gene sequence at codon 39. The strategy was first tested in normal...
| Main Authors: | , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Ferrata Storti Foundation
2007-01-01
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| Series: | Haematologica |
| Online Access: | https://haematologica.org/article/view/4290 |