Lentivector基因疗法治疗X连锁严重联合免疫缺陷

2019-06-12 不详 网络

SIRION公司近日宣布,美国国家过敏和传染病研究所(NIAID)将与SIRION签署许可协议,授权SIRION的LentiBOOST™技术,以提高Lentivector基因疗法治疗X连锁严重联合免疫缺陷的有效性。

SIRION公司近日宣布,美国国家过敏和传染病研究所(NIAID)将与SIRION签署许可协议,授权SIRIONLentiBOOST™技术,以提高Lentivector基因疗法治疗X连锁严重联合免疫缺陷的有效性。

X连锁严重联合免疫缺陷(SCID-X1)是由单个基因ILR2G突变引起的遗传性疾病。该突变导致T细胞、B细胞和自然杀伤(NK)细胞的严重损失,导致受影响儿童的免疫系统的严重损伤。出生时患有这种严重免疫缺陷的婴儿需要在婴儿期进行挽救生命的造血干细胞移植,但大多数患儿仅实现部分免疫重建。直到2016年,Lentivector基因疗法治疗以表达健康的ILR2G时,这一问题才得到逐步解决。


原始出处:

http://www.firstwordpharma.com/node/1646754#axzz5qOw0cfEI

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    2020-04-25 gao_jian4217
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    2019-06-12 坚强007

    向挑战病魔的科研人员致敬!向挑战病魔的科研人员致敬

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