M13 phage based gene therapy platform
AssigneeMassachusetts Institute of Technology
InventorsUyanga Tsedev, Angela M. Belcher
Life of this patent
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Abstract
An engineered phage-derived particle (PDP) for expressing a transgene in a target cell transduced with a bacteriophage, the PDP includes (i) less than about 500 bp of DNA from the bacteriophage genome, (ii) an ITR-flanked therapeutic gene up to 20 kb, (iii) an endosomal escape sequence, (iv) a nuclear localization sequence, and (v) a cell-specific targeting moiety. The PDP may escape lysosomal degradation, traffic across the nuclear envelope and expressed a therapeutic gene in a mammalian cell.
Claims38
An engineered phage-derived particle (PDP) for expressing a transgene in a target cell transduced with a bacteriophage, the PDP comprising:
- (i) less than about 500 bp of DNA from the bacteriophage genome, (ii) an ITR-flanked therapeutic gene up to 20 kb, (iii) an endosomal escape sequence, (iv) a nuclear localization sequence, and (v) a cell-specific targeting moiety, wherein the PDP escapes lysosomal degradation, traffics across the nuclear envelope and expresses a therapeutic gene in a mammalian cell.
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A pharmaceutical composition comprising the recombinant phage particle produced by the method of claim 52, and a pharmaceutically acceptable vehicle.
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Dependencies are inferred from the claim text.