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局部基因疗法KB105治疗转谷氨酰胺酶-1缺乏常染色体隐性遗传性先天性鱼鳞病

2019-06-16 Allan MedSci原创

转谷氨酰胺酶-1KB105基因疗法
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Krystal是一家开发皮肤病药物的基因治疗公司，近日宣布它已向美国FDA提交了一份研究性新药（IND）申请，以启动KB105的I/II期临床试验，KB105是一种基于HSV-1的基因疗法，旨在为患有转谷氨酰胺酶-1（TGM1）缺陷的常染色体隐性遗传性先天性鱼鳞病（ARCI）的患者提供TGM1基因。

Krystal是一家开发皮肤病药物的基因治疗公司，近日宣布它已向美国FDA提交了一份研究性新药（IND）申请，以启动KB105的I/II期临床试验，KB105是一种基于HSV-1的基因疗法，旨在为患有转谷氨酰胺酶-1（TGM1）缺陷的常染色体隐性遗传性先天性鱼鳞病（ARCI）的患者提供TGM1基因。

Krystal利用其皮肤靶向递送（STAR-D）平台技术，将TGM1基因传递给分裂和非分裂的皮肤细胞，使其能够产生该患者缺乏的TGM1蛋白。


原始出处：

http://www.firstwordpharma.com/node/1647663#axzz5r0RYRQBl

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    2019-10-25 yhy100200

    #常染色体#

    0

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    2020-02-02 般若傻瓜

    #遗传性#

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    2019-06-18 新生儿张玉军

    #染色体#

    0

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    2019-06-18 huirong

    #先天性#

    0

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    2019-06-18 chg122

    #局部#

    0

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