首页> 外国专利> COMPOSITIONS AND METHODS FOR IMMUNE TOLERANCE INDUCTION TO FACTOR VIII REPLACEMENT THERAPIES IN SUBJECTS WITH HEMOPHILIA A

COMPOSITIONS AND METHODS FOR IMMUNE TOLERANCE INDUCTION TO FACTOR VIII REPLACEMENT THERAPIES IN SUBJECTS WITH HEMOPHILIA A

机译:甲型血友病患者诱发因子VIII替代疗法的免疫耐受的组合物和方法

摘要

This disclosure relates to tolerance inducing peptide (TIP) derived from the amino acid reference locus (AARL) within a FVIII replacement product (FVIIIrp) based on the differences between the expression product of a subject's F8 gene (sFVIII) and the FVIIIrp to provide tolerance induction before, during, and/or after a FVIII replacement therapy in a subject suffering from Hemophila A. Methods of deriving, making, and using the TIP are also disclosed. In some embodiments, the TIP is associated with a nanoparticle, e.g., PLGA or PLGA-PEMA nanoparticle.
机译:本公开涉及基于受试者的F8基因(sFVIII)和FVIIIrp的表达产物之间的差异,源自FVIII替代产物(FVIIIrp)内的氨基酸参考基因座(AARL)的耐受诱导肽(TIP)。在患有血友病A的受试者中,在FVIII替代疗法之前,期间和/或之后进行诱导。还公开了衍生,制造和使用TIP的方法。在一些实施方案中,TIP与纳米颗粒例如PLGA或PLGA-PEMA纳米颗粒缔合。

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