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AssigneeThe Trustees of the University of Pennsylvania
InventorsJames G Hecker, Micheal Nantz
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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.
A method for non-viral delivery of 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 mono- or poly-peptide when operably-linked to a promoter, the method comprising:
A non-viral method for effecting the rapid, transient expression of nucleic acids in 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 mono- or polypeptide when it is operably-linked to a promoter, the method comprising:
The method of claim 15, wherein the therapeutic polypeptide is effective for preventing or decreasing the severity of neural or nervous system injury, neural damage associated with stroke, traumatic brain injury (TBI), spinal cord injury (SCI), or secondary injury following stroke, brain or spinal cord trauma, neurodegenerative disease, including Alzheimer's disease, Parkinson's disease, Amyotrophic lateral sclerosis, and multiple sclerosis, or medical procedures with a risk of ischemia, hypoxia, hypotension or hyperthermia, including neurosurgical (brain) resections, aortic aneurysm procedures, cardiac bypass procedures, and carotid endarterectomy;
A method for protecting a nucleic acid for intrathecal delivery comprising forming a complex of the nucleic acid with a cationic, lipid congener to form a non-viral, cationic lipid-based delivery vector, wherein the nucleic acid encodes a mono- or poly peptide when it is operably-linked to a promoter, wherein the nucleic acid is protected and stabilized, and wherein the protected, stable nucleic acid remains capable of expressing the encoded polypeptide in the presence of endogenous nucleases.
Dependencies are inferred from the claim text.