Cell-targeting nanoparticles comprising polynucleotide agents and uses thereof

摘要

A method of generating a particle is disclosed, the particle being for delivery of a polynucleotide to a target cell. The method comprises (a) contacting the polynucleotide with a composition comprising cationic molecules, wherein the cationic molecules condense the polynucleotide by electrostatic interactions to generate a complex, wherein the cationic molecules are not comprised in a liposome; and (b) covalently binding the complex to a targeting moiety at a pH equal to or below about 4.5, thereby generating the particle for delivery of the polynucleotide agent to the target cell. Use of the particles and compositions comprising same are also disclosed.

主权项

1. A particle for delivery of a polynucleotide to a target cell, generated according to a method comprising:
(a) contacting the polynucleotide with a composition comprising 1,2-Dilauroyl-sn-Glicero-3-Phosphoethanolamine (DLPE) and 1,2-Dilauroyl-sn-Glicero-3-Glycerol (DLPG), wherein said DLPE and DLPG condense the polynucleotide by electrostatic interactions to generate a complex, and wherein said DLPE and DLPG are not comprised in a liposome; and (b) covalently binding said complex to a glycosaminoglycan targeting moiety at a pH equal to or below about 4.5, thereby generating the particle for delivery of the polynucleotide agent to the target cell.