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OPTIMIZED LENTIVIRAL VECTOR COMPROMISING MINIMAL ENHANCER ELEMENTS FOR STEM CELL GENE THERAPY OF HEMOGLOBINOPATHIES
OPTIMIZED LENTIVIRAL VECTOR COMPROMISING MINIMAL ENHANCER ELEMENTS FOR STEM CELL GENE THERAPY OF HEMOGLOBINOPATHIES
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机译:优化的慢病毒载体损害血吸虫病的干细胞基因治疗的最小增强剂元素
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摘要
In certain embodiments a lentiviral vector having optimized reduced size LCR with improved enhancer activity is provided. In certain embodiments direct treatment of a subject by direct introduction of the vector(s) described herein is contemplated. The lentiviral compositions may be formulated for delivery by any available route including, but not limited to parenteral (e.g., intravenous), intradermal, subcutaneous, oral (e.g., inhalation), transdermal (topical), transmucosal, rectal, and vaginal.
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