Aav-based gene therapy for multiple sclerosis
AssigneeUniversity of Florida Research Foundation, Inc.
InventorBrad E. Hoffman
Life of this patent
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Abstract
Disclosed are AAV viral-based vector compositions useful in delivering a variety of nucleic segments, including those encoding therapeutic polypetides to selected mammalian host cells for use in therapeutic autoimmune modalities, including, for example, the in vivo induction of immunological tolerance via a liver-directed AAV-based gene therapeutic regimen for treating and/or ameliorating autoimmune disorders such as multiple sclerosis.
Claims22
-12. (canceled)
(canceled)
A method for treating a mammal in need thereof comprising systemically administering to the mammal an amount of a particle comprising a recombinant adeno-associated viral (rAAV) nucleic acid vector, the vector comprising a polynucleotide that comprises a nucleic acid segment that encodes a first autoimmune disease therapeutic molecule operably linked to a promoter that is capable of expressing the nucleic acid segment in one or more cells of a mammalian liver, for a time effective to provide the mammal with a therapeutically-effective amount of the therapeutic molecule, wherein the therapeutic molecule comprises a mammalian myelin basic protein (MBP), a proteolipid protein (PLP), a myelin oligodendrocyte glycoprotein (MOG), or a combination thereof.
A method for preventing an autoimmune disease or inhibiting progression of the disease in a mammal, the method comprising:
- systemically administering to the mammal a particle comprising a recombinant adeno-associated viral (rAAV) nucleic acid vector, the vector comprising a polynucleotide that comprises a nucleic acid segment that encodes a first autoimmune disease therapeutic molecule operably linked to a promoter that is capable of expressing the nucleic acid segment in one or more cells of a mammalian liver, in an amount and for a time sufficient to prevent or inhibit progression of the autoimmune disease in the mammal, wherein the therapeutic molecule comprises a mammalian myelin basic protein (MBP), a proteolipid protein (PLP), a myelin oligodendrocyte glycoprotein (MOG), or a combination thereof.
Dependencies are inferred from the claim text.