Paul A Burke1, Suzie H Pun2, Theresa M Reineke3. 1. Burke Bioventures LLC, PO Box 15703, Boston, MA 02215 ; Department of Bioengineering, University of Washington, 3720 15 Ave NE, Box 355061, Seattle, Washington 98195, United States. 2. Department of Bioengineering, University of Washington, 3720 15 Ave NE, Box 355061, Seattle, Washington 98195, United States ; Molecular Engineering & Sciences Institute, University of Washington, 3720 15 Ave NE, Box 355061, Seattle, Washington 98195, United States. 3. Department of Chemistry, University of Minnesota, 207 Pleasant St. SE, Minneapolis, MN 55455.
Abstract
Nucleic acid therapeutics are attracting renewed interest due to recent clinical advances and product approvals. Most leading programs use chemical conjugates, or viral vectors in the case of gene therapy, while several use no delivery system at all. Polymer systems, which have been at the periphery of this renaissance, often involve greater molecular complexity than competing approaches, which must be justified by their advantages. Advanced analytical methods, along with biological tools for characterizing biotransformation and intracellular trafficking, are increasingly being applied to nucleic acid delivery systems including those based on polymers. These frontiers of investigation create the opportunity for an era where highly defined polymer compositions are optimized based on mechanistic insights in a way that has not been previously possible, offering the prospect of greater differentiation from alternatives. This will require integrated collaboration between polymer scientists and those from other disciplines.
n class="Chemical">Nucleic acid therapeutics are apan> class="Gene">ttracting renewed interest due to recent clinical advances and product approvals. Most leading programs use chemical conjugates, or viral vectors in the case of gene therapy, while several use no delivery system at all. Polymer systems, which have been at the periphery of this renaissance, often involve greater molecular complexity than competing approaches, which must be justified by their advantages. Advanced analytical methods, along with biological tools for characterizing biotransformation and intracellular trafficking, are increasingly being applied to nucleic acid delivery systems including those based on polymers. These frontiers of investigation create the opportunity for an era where highly defined polymer compositions are optimized based on mechanistic insights in a way that has not been previously possible, offering the prospect of greater differentiation from alternatives. This will require integrated collaboration between polymer scientists and those from other disciplines.
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