Antisense oligonucleotides materials

Raw material costs for production of antisense oligonucleotide drug substances have been reduced more than 97% since 1991 through process optimization, scale-up economies, and reduced raw material costs,1,60 while the manufacturing and control associated labor costs have fallen even more precipitously. Key changes have converted the process from use of costly... 

Poly(ether thioether)s, poly(ether sulfoxide)s or PES nucleic acids have been suggested as mimics, for natural materials, i.e., with the action of an antisense oligonucleotide. The backbone may bear a plurality of ligands including naturally occurring nucleobases. 

Antisense oligonucleotides are parts of the genetic material that can be used to block the expression of certain genes. For example, a... 

Modification of the Phosphodiester Backbone. Oligonucleotides having modified phosphate backbones have been extensively studied (46). Because altering the backbone makes derivatives generally more resistant to degradation by cellular nucleases, these materials have the potential to be more resilient antisense dmgs. 

Block-graft copolymers synthesized by covalent conjugation of Pluronic and branched PEI were used as materials for preparation of polyplexes. Such polyplexes usually contain three components (i) DNA (ii) Pluronic-PEI conjugate and (iii) free Pluronic (Fig. 7). The formulations were prepared with both plasmid DNA and oligonucleotides (ODN), resulting in stable polyplex dispersion with the particle size in the ranges of 100—200 nm. These polyplexes were used successfully for delivery of plasmid DNA and antisense ODN in vitro... 

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