Nienhuis Arthur W, Nathwani Amit C, Davidoff Andrew M
1 Division of Experimental Hematology, Department of Hematology, St. Jude Children's Research Hospital , Memphis, Tennessee.
2 Department of Haematology, University College London Cancer Institute , London, United Kingdom .
Hum Gene Ther. 2016 Apr;27(4):305-8. doi: 10.1089/hum.2016.018.
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.
腺相关病毒载体已被开发用于治疗甲型和乙型血友病。在对包装载体基因组以产生载体颗粒的细胞进行多质粒转染后，即可获得载体颗粒。迄今为止，已针对乙型血友病进行了三项临床试验。本文描述了这些试验的结果。我们与合作者进行的试验已得出乙型血友病临床疗效的证据。一种治疗甲型血友病的载体已被开发出来，并计划开展一项临床试验。
