Vertex揭示了在首批接受CRISPR/Cas9基因编辑疗法患者中激动人心的数据

2019-11-23 不详 MedSci原创

Vertex及其合作伙伴CRISPR Therapeutics公布了其CRISPR/Cas9基因编辑疗法CTX001治疗的前两名患者的初步阳性数据。令人印象深刻的数据表明,这两名患有严重血红蛋白病,分别为依赖输血的β地中海贫血(TDT)和严重的镰状细胞病(SCD)的患者很可能可以通过该疗法被治愈。

Vertex及其合作伙伴CRISPR Therapeutics公布了其CRISPR/Cas9基因编辑疗法CTX001治疗的前两名患者的初步阳性数据。令人印象深刻的数据表明,这两名患有严重血红蛋白病,分别为依赖输血的β地中海贫血(TDT)和严重的镰状细胞病(SCD)的患者很可能可以通过该疗法被治愈。

TDT患者于2019年第一季度接受了CTX001,该患者的数据反映了9个月的安全性和有效性随访。在参加研究之前,TDT患者每年需要输血16.5次。在CTX001治疗的9个月间,患者是不进行输血的,总血红蛋白水平为11.9g/dL,10.1g/dL胎儿血红蛋白和99.8%的F细胞。

在参加研究之前,SCD患者每年经历7次血管闭塞危机(VOC)。CTX001输注后四个月,患者无VOC,总血红蛋白水平为11.3g/dL,胎儿血红蛋白为46.6%,F细胞为94.7%。

但是,两名患者在用CTX001治疗期间均出现了副作用,但主要研究者并未将这些不良事件视为与研究治疗有关。副作用被认为与接受CRISPR治疗的准备有关,患者必须先接受化疗以破坏其旧的骨髓细胞,这可能会引起不良事件。

Vertex首席执行官Jeffrey Leiden说:"我们今天宣布的数据令人瞩目,并证明CTX001有潜力成为镰刀状细胞病和β地中海贫血患者基于CRISPR/Cas9的治愈性基因编辑疗法。"

"我们期待在未来数月和数年中继续与医生,患者,护理人员和家属合作,为这两种严重疾病提供最佳的治疗方法,并继续加快我们对其他严重疾病的计划,例如杜兴肌营养不良和1型肌强直性营养不良。"

所述CRISPR/CAS-9基因的编辑方法可实现精确编辑活细胞上的靶DNA序列。该技术在DNA内的特定位置引入了断裂,并触发了自我修复机制。进而CRISPR充当了可用于修饰序列的新模板代替恢复原始序列。

原始出处:


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    2019-11-25 yuandd
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    2019-11-25 respect
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    2019-11-25 liuli5080
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    2019-11-25 yaanren
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    2019-11-23 一个字-牛

    学习了谢谢分享

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