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Treatment of the beta-globinopathies through inhibition of RIOK3 activity

Disorders of adult beta-globin synthesis, which include sickle cell disease (SCD) and beta-thalassemia, are the most common monogenic disorders in the world. While the curative potential of bone marrow transplantation has been demonstrated, this approach is limited to a small fraction of affected patients due to the requirement for an HLA-matched donor,...

Published: 4/8/2024 | Inventor(s): John Tisdale, Bjorg Gudmundsdottir, Laxminath Tumburu

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Category(s): Application > Therapeutics, Collaboration Sought > Licensing, TherapeuticArea > Immunology, ResearchProducts > Plasmids/Vectors