Sickle Cell Anemia
Investigators at Children's Hospital target sickle cell anemia
Published in Gene Therapy Weekly, April 16th, 2009
"Increased levels of red cell fetal hemogloblin, whether due to hereditary persistence of expression or from induction with hydroxyurea therapy, effectively ameliorate sickle cell disease (SCD). Therefore, we developed erythroid-specific, gamma-globin lentiviral vectors for hematopoietic stem cell (HSC)-targeted gene therapy with the goal of permanently increasing fetal hemoglobin (HbF) production in sickle red cells," scientists writing in the journal Molecular Therapy report.
"We evaluated two different gamma-globin lentiviral vectors for therapeutic efficacy in the BERK sickle cell mouse model. The first vector, V5, contained the gamma-globin gene driven by...
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Source: Gene Therapy Weekly (2009-04-16)
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