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Xu et al., 2000 - Google Patents

Activation of human γ-globin gene expression via triplex-forming oligonucleotide (TFO)-directed mutations in the γ-globin gene 5′ flanking region

Xu et al., 2000

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Document ID
12984338964405206003
Author
Xu X
Glazer P
Wang G
Publication year
Publication venue
Gene

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Snippet

Human β-globin disorders, such as sickle cell anemia and β-thalassemia, are relatively common genetic diseases cause by mutations in the β-globin gene. Increasing γ-globin gene expression has been found to greatly reduce the disease symptom. However, the γ …
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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    • C12Q2600/00—Oligonucleotides characterized by their use

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