| 摘要 |
The present invention relates to management and treatment of hemoglobinopathies, such as sickle cell anemia and beta -thalassemia. The invention also relates to developing research animals and cell lines for the study of hemoglobinopathies and their therapies. The invention utilizes third strand oligonucleotides to target double-stranded nucleic acid sequences in or near the globin genes or in or near sequences controlling expression of those genes to cause either a desired mutation or nucleic damage to stimulate homologous recombination with a supplied donor nucleic acid.
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