Device and method for encapsulated gene therapy
First Claim
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1. A biocompatible capsule comprising:
- a core comprising living packaging cells that secrete a viral vector for infection of a target cell, wherein the viral vector is a retroviral vector, the vector comprising a heterologous gene encoding a biologically active molecule operably linked to a promoter that regulates expression of the biologically active molecule in the target cell, the heterologous gene being no more than 8 kb in length; and
an external jacket surrounding said core, said jacket comprising a permeable biocompatible material, said material having a porosity selected to permit passage of retroviral vectors of 100 nm diameter thereacross, permitting release of said viral vector from said capsule.
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Abstract
Methods and devices are provided for gene therapy using encapsulated packaging cell lines to deliver viral particles carrying at least one heterologous gene encoding at least one biologically active molecule.
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Citations
21 Claims
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1. A biocompatible capsule comprising:
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a core comprising living packaging cells that secrete a viral vector for infection of a target cell, wherein the viral vector is a retroviral vector, the vector comprising a heterologous gene encoding a biologically active molecule operably linked to a promoter that regulates expression of the biologically active molecule in the target cell, the heterologous gene being no more than 8 kb in length; and an external jacket surrounding said core, said jacket comprising a permeable biocompatible material, said material having a porosity selected to permit passage of retroviral vectors of 100 nm diameter thereacross, permitting release of said viral vector from said capsule. - View Dependent Claims (2, 3, 4, 5)
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6. A method for making a biocompatible capsule for delivery of a viral vector, comprising the steps of:
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selecting a jacket, the jacket comprising a biocompatible material having a porosity selected to permit passage of retroviral vectors of 100 nm diameter thereacross, permitting release of the retroviral vector, loading packaging cells into the jacket, the packaging cells secreting said viral vector comprising a heterologous gene encoding at least one biologically active molecule operably linked to a promoter that regulates expression of the biologically active molecule in the target cell and being no more than 8 kb in length; and sealing the jacket to encapsulate the packaging cells.
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7. A method of delivering a viral vector to a cell in vitro by placing at least one capsule in proximity with the cell, said capsule comprising:
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a core comprising living packaging cells that secrete the viral vector, wherein the viral vector is a retroviral vector, the vector comprising a heterologous gene encoding a biologically active molecule operably linked to a promoter that regulates expression of the biologically active molecule in said cell, the heterologous gene being no more than 8 kb in length; and an external jacket surrounding said core, said jacket comprising a biocompatible material, said material having a porosity selected to permit passage of retroviral vectors of 100 nm diameter thereacross, permitting release of the viral vector from the capsule. - View Dependent Claims (8, 9, 10, 11, 12, 13, 14, 15)
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16. A method of delivering a viral vector to a cell by placing at least one capsule in proximity with the cell, said capsule comprising:
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a core comprising living packaging cells that secrete the viral vector, wherein the viral vector is a retroviral vector, the vector comprising a heterologous gene encoding a biologically active molecule operably linked to a promoter that regulates expression of the biologically active molecule in said cell, wherein the heterologous gene is HSV-tk; and an external jacket surrounding said core, said jacket comprising a biocompatible material, said material having a porosity selected to permit passage of retroviral vectors of 100 nm diameter thereacross, permitting release of the viral vector from the capsule. - View Dependent Claims (17, 18, 19, 20, 21)
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Specification