首页>
外国专利>
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
展开▼
机译:血红蛋白病干细胞基因治疗中优化的微病毒载体包含最小的增强子
展开▼
页面导航
摘要
著录项
相似文献
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.
展开▼