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OwnerIan Alexander, Sharon Cunningham +1inventors
InventorsIan Alexander, Sharon Cunningham, Andras Nagy
Estimating the term…
Provided herein are methods for facilitating or inducing stable transgene integration and expression in a proliferating cell, comprising administering to the cell (i) a recombinant AAV (rAAV) vector comprising the transgene flanked by transposon-derived inverted terminal repeat sequences, which sequences are in turn flanked by AAV-derived inverted terminal repeat regions, and (ii) a source of a transposase that recognises said transposon-derived inverted terminal repeat sequences and directs the genomic integration of the transgene into the genome of the proliferating cell. Also provided are methods and transgene delivery systems for the treatment or prevention of diseases affecting, associated with or characterised by proliferating cells, including paediatric liver diseases, bone marrow diseases and cancer.
A method for stably integrating a transgene into the genome of a proliferating cell and/or inducing stable transgene expression in a proliferating cell, the method comprising administering to the cell:
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A method for treating a disease of, affecting, or associated with, a proliferating cell, comprising administering to a subject in need thereof (i) a recombinant AAV (rAAV) vector comprising a transgene flanked by transposon-derived inverted terminal repeat sequences, which sequences are in turn flanked by AAV-derived inverted terminal repeat regions;
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A transgene delivery and expression system for inducing stable transgene expression in a proliferating cell or for treating a disease of, affecting, or associated with, a proliterating cell, wherein the system comprises (i) a recombinant AAV (rAAV) vector comprising the transgene flanked by transposon-derived inverted terminal repeat sequences, which sequences are in turn flanked by AAV-derived inverted terminal repeat regions;
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Dependencies are inferred from the claim text.