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    • 5. 发明申请
    • INJECTION OF SINGLE-STRANDED OR SELF-COMPLEMENTARY ADENO-ASSOCIATED VIRUS 9 INTO THE CEREBROSPINAL FLUID
    • 单束或自补体腺相关病毒注入脑脊液
    • WO2017136202A1
    • 2017-08-10
    • PCT/US2017/014914
    • 2017-01-25
    • EMORY UNIVERSITYREGENXBIO INC.
    • DONSANTE, AnthonyKOZARSKY, Karen
    • C12N15/63C12N15/861A61K48/00
    • C12N15/86A61K48/0075C12N2750/14143
    • It is disclosed herein that ssAAV and scAAV vectors of the same serotype administered by injection into the cerebrospinal fluid (CSF) via the intracerebroventricular (ICV) or intrathecal (cisternal or lumbar) route exhibit different cellular tropisms in the central nervous system. Thus, a subject can be treated by injection into the CSF of ssAAV or scAAV vector encoding a therapeutic protein, such as an ssAAV9 or scAAV9 vector. The therapeutic protein can be targeted to specific cells using these vectors. In some embodiments, scAAV9 is utilized to achieve superior transduction in the hippocampus, cerebellum and cerebral cortex where both neurons, particularly Purkinje neurons, and glial cells (such as astrocytes) are transduced. In other embodiments, ssAAV9 is utilized to minimize transduction of astrocytes. In further embodiments, an immunosuppressive agent is also administered to the subject.
    • 本文中公开了通过脑室内(ICV)或鞘内(脑池或腰椎)途径注射到脑脊液(CSF)中的相同血清型的ssAAV和scAAV载体显示不同的细胞向性 中枢神经系统。 因此,可以通过向CSF中注射编码治疗性蛋白质如ssAAV9或scAAV9载体的ssAAV或scAAV载体来治疗受试者。 使用这些载体可将治疗性蛋白质靶向特定细胞。 在一些实施方案中,利用scAAV9在海马,小脑和大脑皮层中实现优异的转导,其中神经元,特别是浦肯野神经元和神经胶质细胞(例如星形胶质细胞)都被转导。 在其他实施方案中,利用ssAAV9来最小化星形胶质细胞的转导。 在进一步的实施方案中,还将免疫抑制剂施用给受试者。