Methods for treating articular disease or dysfunction using self-complimentary adeno-associated viral vectors
AssigneesUniversity of Florida Research Foundation, Inc., The Brigham and Women's Hospital, Inc.
InventorsSteven Craig Ghivizzani, Christopher Howard Evans, Elvire Gouze, Jean-Noel Gouze
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Abstract
Disclosed are methods and compositions for treating articular diseases such as osteoarthritis and rheumatoid arthritis, or articular dysfunction in mammals. Also disclosed are self-complimentary AAV vectors useful in gene therapy and delivery of therapeutic constructs to mammalian cells, and in particular, articular joints, cartilage, ligaments, tendons, and surrounding tissues.
Claims25
A method for providing to a mammal in need thereof, a therapeutically-effective amount of a mammalian Interleukin-1 Receptor Antagonist peptide or polypeptide, said method comprising at least the step of:
- (a) introducing into a plurality of cells or in a selected tissue of said mammal, a composition comprising a recombinant self-complimentary adeno-associated viral vector, wherein said vector comprises a nucleic acid segment that encodes a biologically-active mammalian IL-1Ra peptide or polypeptide, operably linked to a promoter that is capable of expressing said nucleic acid segment in a mammalian host cell that comprises said vector, in an amount, and for a time effective, to provide said mammal with said therapeutically-effective amount of said mammalian Interleukin-1 Receptor Antagonist peptide or polypeptide.
A method for providing directly to a mammalian joint or periarticular space, a therapeutically-effective amount of a biologically-active IL-1Ra peptide or polypeptide, said method comprising directly injecting into at least a first joint or periarticular space of said mammal an effective amount of a composition comprising:
- a recombinant self-complimentary adeno-associated viral vector that comprises a nucleic acid segment that encodes a biologically-active mammalian IL-1Ra peptide or polypeptide, operably linked to a promoter that is capable of expressing said nucleic acid segment in a mammalian host cell that comprises said vector, in an amount, and for a time effective, to directly provide to said mammalian joint or periarticular space, said therapeutically-effective amount of said mammalian Interleukin-1 Receptor Antagonist peptide or polypeptide.
A method for treating or ameliorating the symptoms of an articular defect, injury, disease or dysfunction in a mammal, said method comprising administering to said mammal a composition comprising:
- recombinant self-complimentary adeno-associated viral vector that comprises a nucleic acid segment encoding a biologically-active mammalian IL-1Ra peptide or polypeptide, said segment operably linked to at least a first promoter that is capable of expressing said nucleic acid segment in a mammalian host cell that comprises said vector;
- in an amount and for a time sufficient to treat or ameliorate the symptoms of said articular defect, injury, disease or dysfunction in said mammal.
Dependencies are inferred from the claim text.