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寡核苷酸疗法
寡核苷酸疗法

基因治疗为彻底治愈带来了希望

寡核苷酸疗法

基因疗法为彻底治愈带来了希望。这种疗法在被广泛应用之前还需要克服很多挑战。

寡核苷酸(OGN)疗法逐渐被认为是治愈疾病的一种重要治疗方式。然而,与大多数生物疗法不同的是,OGN药物的开发往往需要对血液和组织等多种复杂的生物基质进行高灵敏度和特异性测定,以测量其暴露量和分布,从而了解PK、PD和安全性。

在方达我们已经开发出了高度灵敏和特异性的定量方法,使用杂交免疫测定法和LC-MS技术测定血浆和组织中的OGN。在本网络研讨会中,我们将介绍OGN定量方法的开发,验证和这些技术的比较,以及反义寡核苷酸的案例研究。

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