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New gene therapy vector promising for sickle cell, beta-thalassemia


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Journal
Molecular Therapy
Reuters Health - 10/02/2021 - A new lentiviral gene therapy vector, ALS20, triggers more hemoglobin production at a lower dose than vectors currently used in trials of blood disorders such as sickle cell disease and beta-thalassemia, researchers say. "We hope our vector will be curative, (while) requiring less integration per genome than current vectors," Drs. Laura Breda and Stefano Rivella of Children's Hospital of Philadelphia told Reuters Health by email "If this is the case, we would like to evaluate novel approaches using reduced conditioning regimens and, eventually, direct delivery of the vector to the bone marrow of patients, bypassing myeloablation." Currently, gene therapy trials for beta-globinopathies involve transferring the beta-globin gene, which requires integration of three to four copies per genome in most target cells. "This high proviral load may increase genome toxicity, potentially limiting the safety of this therapy and relegating its use to ...


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