Worldwide use of factor IX Padua for hemophilia B gene therapy.
Worldwide use of factor IX Padua for hemophilia B gene therapy.
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全球范围内使用 IX 因子帕多瓦进行血友病 B 基因治疗。
DOI：
10.1016/j.ymthe.2022.06.002
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发表时间：
2022
期刊：
影响因子：
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通讯作者：
Samelson-Jones,BenjaminJ
中科院分区：
文献类型：
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作者：
Samelson-Jones,BenjaminJ
薛等人的遭遇是苦乐参半的巧合。几乎就在FIX-Padua的共同发现者2 Valder R. Arruda在短暂患病后意外去世的同一天，Lancet Hematology 1在线发表了他们利用高特异性活性因子IX (FIX)变体Padua对中国B型血友病(HB)男性成功进行腺相关病毒(AAV)基因治疗试验。他们的报告增加了越来越多有前途的 HB 基因治疗产品清单，所有产品均使用 FIX-Padua, 3, 4，其中至少有 3 个处于第 3 阶段。Arruda 博士在一定程度上是由于他早期在祖国巴西从事血液学的经历，他一直对解决西方国家以外全球 80% 的血友病人群获得治疗机会有限的问题感兴趣； 5 因此，在基因治疗后，表达 FIX-Padua 的 10 名非西方 HB 受试者中至少有 9 名的表型治愈，这是对他在基因治疗领域的贡献的及时证明。费城 Arruda 博士的实验室和意大利帕多瓦 Paolo Simioni 博士的团队合作，FIX-Padua 被鉴定为一种罕见的 X 连锁血栓形成倾向。 2 这两位合作者 10 年前就认识了，当时他们都是荷兰莱顿的博士后，这体现了 Arruda 博士将长期研究与
It is a bittersweet coincidence the Xue et al. published online their successful adenoassociated virus (AAV) gene therapy trial for Chinese men with hemophilia B (HB) utilizing the high-specific-activity factor IX (FIX) variant Padua in Lancet Hematology 1 almost on the same day that the co-discoverer of FIX-Padua, 2 Valder R. Arruda, passed away unexpectedly after a brief illness. Their report augments a growing list of promising gene therapy products for HB, all using FIX-Padua, 3, 4 including at least three in phase 3. In part because of his early experiences practicing hematology in his native Brazil, Dr. Arruda was always interested in addressing the 80% of the worldwide hemophilia population outside of Western countries that have limited access to treatments; 5 thus, the phenotypic cure of at least 9 out of 10 non-Western HB subjects expressing FIX-Padua after gene therapy is a well-timed testament to his contributions to the field of gene therapy.FIX-Padua was identified as a rare X-linked thrombophilia in a collaboration between Dr. Arruda’s laboratory in Philadelphia and Dr. Paolo Simioni’s team in Padua, Italy. 2 The two collaborators had met 10 years prior when they were both post-docs in Leiden, the Netherlands, which was typical of Dr. Arruda’s ability to mix long-