美国Avigen公司的研究者开发了一项基因治疗技术,即在小鼠中长期表达人红细胞生成素(EPO).此项研究是用Avigen公司的腺病毒伴随病毒(AAV)载体将EPO基因引入小鼠,EPO是一种能刺激红细胞生成的蛋白质.单剂肌肉注射携带EPO基因的AAV载体能使小鼠血清中的人EPO水平升高.并增高红细胞水平.这些水平在6周内逐渐升高,并在此后8个月中保持稳定.此项研究除了证实AAV载体的效力外,还显示了EPO基因治疗的潜力,它可取代EPO用于治疗肾功能衰竭、艾滋病或化疗引起的贫血. Researchers at Avigen Inc. in the United States have developed a gene therapy technique that involves the long-term expression of human erythropoietin (EPO) in mice using the Avigen adenovirus-associated virus (AAV) vector to introduce the EPO gene into small In rats, EPO is a protein that stimulates erythropoiesis.A single intramuscular injection of the AAV vector carrying the EPO gene increases the level of human EPO in mouse serum and increases the level of erythrocytes, which gradually increases within 6 weeks , And remained stable over the next eight months.6 This study, in addition to confirming the efficacy of AAV vectors, shows the potential of EPO gene therapy to replace EPO for the treatment of renal failure, AIDS or chemotherapy-induced anemia.