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基因疗法OTL-101治疗ADA-SCID的可行性

2019-02-24 不详 网络

OTL-101基因疗法ADA-SCID
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Orchard Therapeutics是一家领先的生物制药公司，致力于通过创新的基因疗法改变患有严重和危及生命的罕见疾病，近日Orchard将公布来自登记试验的20名患者的随访数据，该试验评估了OTL-101（一种自体、离体、造血干细胞基因疗法）用于治疗因腺苷脱氨酶缺乏引起的严重联合免疫缺陷（ADA-SCID）的可行性。

Orchard Therapeutics是一家领先的生物制药公司，致力于通过创新的基因疗法改变患有严重和危及生命的罕见疾病，近日Orchard将公布来自登记试验的20名患者的随访数据，该试验评估了OTL-101（一种自体、离体、造血干细胞基因疗法）用于治疗因腺苷脱氨酶缺乏引起的严重联合免疫缺陷（ADA-SCID）的可行性。这些随访数据将在德克萨斯州休斯顿举行的2019年移植和细胞治疗会议上讨论。

ADA-SCID是一种罕见的，危及生命的遗传性疾病，由ADA基因突变引起。ADA酶的缺陷导致免疫系统细胞功能障碍，包括B、T和自然杀伤细胞的功能障碍。患有ADA-SCID的患者无法抵抗并经常死于细菌、病毒和真菌感染。

加州大学洛杉矶分校微生物学、免疫学和分子遗传学教授，该研究的主要研究者Donald B. Kohn博士说：“随着一些患者随访的进行，与历史对照组（接受造血干细胞移植）相比，OTL-101治疗ADA-SCID患者的整体和无事件生存率持续良好。我们对这些结果非常兴奋，并期待为这种严重且危及生命的疾病推出变革性的治疗方案”。


原始出处：

http://www.firstwordpharma.com/node/1626480#axzz5gKHwd300

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    2019-12-21 ms49895894946433513521

    #OTL-101#

    0

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    2019-10-01 xlxchina

    #CID#

    0

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    2020-01-03 xugumin

    #SCID#

    0

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    2019-06-16 循证小兵

    #可行性#

    0

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    2019-02-26 般若傻瓜

    #ADA#

    0