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US09149543B2 Methods and models for rapid, widespread delivery of genetic material to the CNS using non-viral, cationic lipid-mediated vectors 有权
使用非病毒,阳离子脂质介导的载体快速,广泛传播遗传物质到CNS的方法和模型

Methods and models for rapid, widespread delivery of genetic material to the CNS using non-viral, cationic lipid-mediated vectors
Abstract:
Provided are safe, non-invasive, non-viral delivery methods for providing a nucleic acid into the neuronal and non-neuronal cells of the central nervous system (CNS) of a subject to protect neuronal and non-neuronal cells from ischemic or traumatic injury, wherein the nucleic acid encodes a therapeutic proteins, specifically providing rapid transient expression and widespread distribution for in vitro or in vivo applications. Further provided are methods for the intrathecal delivery to the cerebrospinal fluid (CSF) of a neuroprotective gene sequence, e.g., a heat shock protein (HSP), complexed with cationic lipid compositions to achieve such delivery, and the complexes used therein.
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