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FDA批准UniQure开展亨廷顿病的AAV基因治疗临床试验

2019-01-26 MedSci MedSci原创

美国监管机构已批准基因疗法UniQure公司的AMT-130基因疗法应用于罕见遗传性神经退行性疾病亨廷顿病的1/2期临床试验,目前该公司正计划在下半年招募其第一批受试者。

美国监管机构已批准UniQure公司的AMT-130基因疗法应用于罕见遗传性神经退行性疾病亨廷顿病的1/2期临床试验,目前该公司正计划在下半年招募其第一批受试者。

据估计,美国约有25000人患有亨廷顿氏病,该类患者体内特定的DNA序列,称为亨廷顿基因异常不稳定并引起大脑损伤,进而导致不自主运动(舞蹈病)以及认知和记忆问题。

UniQure表示,AMT-130是第一种基于腺相关病毒(AAV)的基因疗法,将被批准用于亨廷顿氏症的试验。它的1/2期研究将测试该疾病患者中单次递增剂量的AMT-130,旨在测试其安全性和耐受性,并提供一些有效性的初步数据。

原始出处:


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    2019-01-28 xuqianhua
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    2019-01-26 坚强007

    向科研人员致敬!!!

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