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An improved fetal hemoglobin for gene correction of sickle cell anemia
An improved fetal hemoglobin for gene correction of sickle cell anemia
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机译:用于镰状细胞性贫血的基因校正的改良胎儿血红蛋白
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摘要
Methods and compositions disclosed herein generally relates to methods of determining minimum hematopoietic stem cell (HSC) chimerism and gene dosage for correction of a hematopoietic disease; in particular, in in vivo models. The invention also relates to modified lentiviral expression vectors for increasing a viral titer and various methods for increasing such titers as well as expression vectors capable of enhancing such titers. The invention also relates to CHS4 chromatin insulator-derived functional insulator sequences. The invention also relates to methods for genetic correction of diseases or reducing symptoms thereof, such as sickle cell anemia or β-thalassemia.
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