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US 2023/0270887 A1Published application
Active delivery of nucleic acids and peptides, methods and uses
OwnerMark ISALAN, Michal MIELCAREKinventors
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Abstract
Disclosed herein are polypeptides for use in treating diseases. Therapeutic uses and methods for treating diseases are also disclosed. Also disclosed is a method and associated peptides and nucleic acids for active, long-term delivery of therapeutic molecules to target cells in vivo or in vitro.
Claims17
An isolated polynucleotide encoding a polypeptide for delivery of an effector peptide to a target cell different to a source cell in which it was expressed;
- the polynucleotide comprising:
- (a) sequence encoding a polypeptide, the polypeptide comprising:
- (i) the effector peptide sequence;
- (ii) a cell secretion peptide sequence operably linked to the effector peptide sequence;
- (iii) a cell penetration and/or cell localisation peptide sequence operably linked to the effector peptide sequence;
- and
- (b) a polypeptide expression element operable to cause the polypeptide to be expressed in the source cell in vivo.
4 more elements
An in vitro or in vivo method for delivery of a biological effector moiety to a target cell different to the cell in which it was expressed, the method comprising:
- (i) providing a nucleic acid expression construct encoding an expressible biological effector peptide, the biological effector peptide adapted for cell secretion from a first target cell and cell penetration of a second target cell, wherein the first and second target cells may be of the same type or of different types;
- (ii) delivering the nucleic acid expression construct to the first target cell;
- (iii) expressing the expressible biological effector peptide in the first target cell and allowing it to be secreted from the first target cell;
- (iv) bringing the secreted biological effector peptide into contact with a second target cell under conditions that allow the biological effector peptide to penetrate the second target cell;
- thereby to deliver the biological effector moiety to the target cell.
2 more elements
Dependencies are inferred from the claim text.