Methods and compositions for therapies using genes encoding secreted proteins
AssigneeBiogen, Inc.
InventorsJames G. Barsoum, Albert Xiao Qin
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Abstract
Methods and pharmaceutical compositions for modifying cells of a mammalian recipient with DNA encoding a secreted protein such as human interferon in situ are provided. The methods include forming a secreted protein expression system in vivo or ex vivo and administering the expression system to the mammalian recipient. The expression system and methods are useful for the localized and systemic delivery of interferons in situ.
Claims37
A cell expression system for in vivo expression of an interferon protein in a cell of a mammalian recipient, the expression system comprising:
- a cell of the same species as the mammalian recipient;
- and an expression vector contained therein for expressing the interferon protein, wherein said expression vector is useful for expressing the interferon protein in a cell for human gene therapy, wherein the recipient has a condition treatable by delivery of the interferon protein to a site of the recipient.
A cell expression system for expressing in a cell of a mammalian recipient in vivo, a secreted protein for treating a condition, the expression system comprising:
- a plurality of cells of the same species as the mammalian recipient;
- and an expression vector contained therein for expressing the secreted protein, wherein said expression vector is contained within only a portion of said plurality of cells, said vector useful for expressing the secreted protein for human gene therapy, wherein the condition is treatable by delivery of the secreted protein to a site of the recipient
A cell expression system comprising a plurality of cancer cells, at least a portion of said plurality of cancer cells containing a viral vector having incorporated therein an isolated polynucleotide encoding, upon expression, an interferon protein.
A composition for delivery of a secreted protein to a site of a mammalian recipient, comprising:
- a plurality of genetically modified cells of the same species as the mammalian recipient, at least a portion of said cells containing an expression vector for expressing an effective amount of the secreted protein in said cells for human gene therapy.
A method for making an ex vivo gene therapy pharmaceutical preparation for administration to a mammalian recipient, the method comprising the steps of:
- (a) forming a plurality of cells of the same species as the mammalian recipient, (b) introducing an expression vector for expressing a secreted protein into at least one cell of said plurality to form at least one genetically modified cell, and wherein the expression vector is useful for expressing the secreted protein in said cells for human gene therapy;
- and (c) placing the at least one genetically modified cell in a pharmaceutically acceptable carrier to obtain a pharmaceutical preparation that is suitable for administration to a site of the mammalian recipient, wherein the secreted protein is for treating a condition that is treatable by delivery of the secreted protein to the cell-compatible site.
A method for gene therapy, which comprises genetically modifying at least one cell of a mammalian recipient via the steps of (a) introducing an expression vector for expressing a secreted protein into the at least one cell to form at least one genetically modified cell, wherein the cell is of the same species as the mammalian recipient, and wherein the expression vector is useful for expressing the secreted protein in the cell for human gene therapy;
- and (b) allowing the genetically modified cell to contact a site of the mammalian recipient, wherein the mammalian recipient has a condition that is treatable by delivery of the secreted protein to the site or by administration of the secreted protein.
A method of ex vivo gene therapy, comprising the steps of
- removing a plurality of cells from a subject;
- administering a recombinant adenovirus to at least one cell of said plurality of cells, such that there exists an excess of cells not containing the adenovirus, wherein the adenovirus has a deletion or mutation in its E1 gene, the adenovirus further comprising an isolated polynucleotide encoding a secreted protein;
- and
- reintroducing said plurality of cells into the subject
A method of in vivo gene therapy, comprising administering a viral vector that includes an isolated polynucleotide encoding human interferon-beta protein directly into a cell of a subject without first removing said cell from the subject.
Dependencies are inferred from the claim text.