Gene Therapy for Hemophilia.
Gene Therapy for Hemophilia.
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血友病的基因治疗。
DOI：
10.1089/hum.2016.018
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发表时间：
2016
影响因子：
4.2
通讯作者：
Nienhuis AW
中科院分区：
文献类型：
--
作者：
Nienhuis AW
已经开发了用于治疗血友病A和B的腺相关病毒载体。在包装载体基因组以产生载体颗粒的细胞的多质粒转染后实现载体颗粒的衍生。迄今为止，已对血友病B进行了三项临床试验。这些试验的结果进行了说明。我们与合作者进行的试验已获得血友病B临床疗效的证据。已经开发了一种治疗血友病A的载体，并计划进行临床试验。
Adeno-associated viral vectors have been developed for the treatment of hemophilia A and B. Derivation of vector particles is achieved after multiplasmid transfection of cells that package the vector genome to yield vector particles. To date, three clinical trials have been performed for hemophilia B. The results of these trials are described. The trial that we conducted with our collaborators has yielded evidence of clinical efficacy for hemophilia B. A vector for treating hemophilia A has been developed and a clinical trial is planned.