NON-VIRAL DNA VECTORS AND USES THEREOF FOR EXPRESSING FVIII THERAPEUTICS
The application describes ceDNA vectors having linear and continuous structure for delivery and expression of a transgene. ceDNA vectors comprise an expression cassette flanked by two ITR sequences, where the expression cassette encodes a transgene encoding FVIII protein. Some ceDNA vectors further...
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Main Authors | , , , |
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Format | Patent |
Language | English French German |
Published |
21.06.2023
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Abstract | The application describes ceDNA vectors having linear and continuous structure for delivery and expression of a transgene. ceDNA vectors comprise an expression cassette flanked by two ITR sequences, where the expression cassette encodes a transgene encoding FVIII protein. Some ceDNA vectors further comprise cis-regulatory elements, including regulatory switches. Further provided herein are methods and cell lines for reliable gene expression of FVIII protein in vitro, ex vivo and in vivo using the ceDNA vectors. Provided herein are method and compositions comprising ceDNA vectors useful for the expression of FVIII protein in a cell, tissue or subject, and methods of treatment of diseases with said ceDNA vectors expressing FVIII protein. Such FVIII protein can be expressed for treating disease, e.g., hemophilia A. |
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AbstractList | The application describes ceDNA vectors having linear and continuous structure for delivery and expression of a transgene. ceDNA vectors comprise an expression cassette flanked by two ITR sequences, where the expression cassette encodes a transgene encoding FVIII protein. Some ceDNA vectors further comprise cis-regulatory elements, including regulatory switches. Further provided herein are methods and cell lines for reliable gene expression of FVIII protein in vitro, ex vivo and in vivo using the ceDNA vectors. Provided herein are method and compositions comprising ceDNA vectors useful for the expression of FVIII protein in a cell, tissue or subject, and methods of treatment of diseases with said ceDNA vectors expressing FVIII protein. Such FVIII protein can be expressed for treating disease, e.g., hemophilia A. |
Author | KERR, Douglas, Anthony KLATTE, Debra SILVER, Nathaniel SAMAYOA, Phillip |
Author_xml | – fullname: SAMAYOA, Phillip – fullname: KLATTE, Debra – fullname: KERR, Douglas, Anthony – fullname: SILVER, Nathaniel |
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DocumentTitleAlternate | NICHT-VIRALE DNA-VEKTOREN UND DEREN VERWENDUNGEN ZUR EXPRESSION VON FVIII-THERAPEUTIKA VECTEURS D'ADN NON VIRAUX ET LEURS UTILISATIONS POUR EXPRIMER DES AGENTS THÉRAPEUTIQUES DU FACTEUR VIII |
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Notes | Application Number: EP20200769344 |
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PublicationYear | 2023 |
RelatedCompanies | Generation Bio Co |
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Snippet | The application describes ceDNA vectors having linear and continuous structure for delivery and expression of a transgene. ceDNA vectors comprise an expression... |
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Title | NON-VIRAL DNA VECTORS AND USES THEREOF FOR EXPRESSING FVIII THERAPEUTICS |
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