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US07901671B2 Therapeutic retroviral vectors for gene therapy 有权
用于基因治疗的治疗性逆转录病毒载体

Therapeutic retroviral vectors for gene therapy
Abstract:
The invention provides improved compositions and methods for achieving gene therapy in hematopoietic cells and hematopoietic precursor cells, including erythrocytes, erythroid progenitors, and embryonic stem cells. The invention further provides improved gene therapy methods for treating hematopoietic-related disorders. Retroviral gene therapy vectors that are optimized for erythroid specific expression and treatment of hemoglobinopathic conditions are disclosed.
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