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×î½üͶÁËһƪ¹ØÓÚ»ùÒòÖÎÁÆ·½ÃæµÄÎÄÕ£¬ÏÂÃæÊÇÉó¸åÈ˸ø³öµÄÒâ¼û£¬¸öÈ˸оõͦ³éÏóµÄ£¬²»ÖªµÀ´ó¼ÒÓÐûÓÐʲôºÃµÄ½¨Òé£¿ÌØ±ðÊǸÃÈçºÎ»Ø´ðÄØ£¿Ð»Ð»¡£ Reviewer #1: The results of the authors demonstrated that rAAV2-mediated ¦Â-globin virus can efficiently mediate the expression of normal ¦Â-globin gene in fetal hematopoietic cells from ¦Â-thalassemia patients, for at least 21-35 days in the recipient mice and the level of human ¦Â-globin expression significantly increased by 1.2-1.8 folds, as compared with that of controls. So, for the final potential use of rAAV-based clinical gene therapy for human ¦Â-thalassemia, more experimental works should be done to answer the questions: ¢ÅActually how are the durability and the stability of the rAAV-based transduction? ¢ÆHow is the level of peripheral hemoglobin after the rAAV-based gene therapy? |
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