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葛兰素史克用于治疗儿童免疫疾病的基因药获欧盟批准

2016-06-05 佚名 中国科技网

据《泰晤士报》近日报道,英国葛兰素史克公司和意大利科学家联合开发的首个用于治疗儿童免疫疾病的基因治疗药物Strimvelis,日前获欧洲药品管理局批准在欧洲销售,这标志着修改错误基因这一先进技术又向前迈出了一步。 从理论上说,基因治疗人体自身细胞能有效纠正一些对健康危害极大的基因突变,但在实践上,研发安全有效的基因治疗方案却面临极大困难。此次获批的Strimvelis主要用于治疗少数患有严重

据《泰晤士报》近日报道,英国葛兰素史克公司和意大利科学家联合开发的首个用于治疗儿童免疫疾病的基因治疗药物Strimvelis,日前获欧洲药品管理局批准在欧洲销售,这标志着修改错误基因这一先进技术又向前迈出了一步。

从理论上说,基因治疗人体自身细胞能有效纠正一些对健康危害极大的基因突变,但在实践上,研发安全有效的基因治疗方案却面临极大困难。此次获批的Strimvelis主要用于治疗少数患有严重联合免疫缺陷病(ADA-SCID)的儿童。SCID有时也被称作“泡泡婴儿(Bubble baby)”疾病,这些婴儿在出生时免疫系统极其脆弱,必须在无菌环境中才能存活,若不治疗,患儿通常会在两岁内面临死亡。该基因治疗是从儿童自身骨髓中提取出干细胞,并替换其中有缺陷的基因,使其可以生产ADA酶,然后再植到患儿体内以修复自身免疫系统。

目前,许多癌症研究表明,大多数癌症(诸如乳腺癌等)是由其他多种疾病组合发生而引起的。葛兰素史克罕见疾病研发中心负责人马丁·安德鲁斯称,基因治疗也许是应对复杂疾病的最佳方法,因为利用人体自身去抵抗疾病的方法被认为是“未来医学发展的方向,也是药学研究的前沿领域”,希望能够将基因治疗方法延伸到更多疾病上去。

Strimvelis是继荷兰UniQure公司基因治疗药物Glybera之后,第2个获批在欧洲销售的基因治疗药物。Glybera主要用于治疗一种罕见的成人血液疾病。迄今,美国积极从事基因治疗药物的生物科技公司日益增多,但美国食品药品监督管理局尚未批准任何基因治疗药物。

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    2016-06-06 milkshark

    这个有意义

    0

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    2016-06-06 milkshark

    多多益善

    0

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    2016-06-05 沉心多思

    不错的文章,多学习

    0

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上周科学家们聚集在美国华盛顿召开了基因编辑年度会议,基因编辑领域目前进展火热,在一些小型的临床试验中大放异彩,展现出非常好的应用前景。现在许多人认为CRISPR这种强大的基因编辑技术将会大大推动基因治疗的发展。但是CRISPR技术真的已经为基因治疗做好准备了吗?Science杂志对该技术的前景和风险进行了报道。 CRISPR如何发挥作用? 传统的基因治疗利用相对暴力的方法进行基因导入:

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