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The advent of genome editing has transformed the therapeutic landscape for several debilitating diseases, and the clinical outlook for gene therapeutics has never been more promising. The therapeutic potential of nucleic acids has been limited by a reliance on engineered viral vectors for delivery. Chemically defined polymers can remediate technological, regulatory, and clinical challenges associated with viral modes of gene delivery. Because of their scalability, versatility, and exquisite tunability, polymers are ideal biomaterial platforms for delivering nucleic acid payloads efficiently while minimizing immune response and cellular toxicity. While polymeric gene delivery has progressed significantly in the past four decades, clinical translation of polymeric vehicles faces several formidable challenges. The aim of our Account is to illustrate diverse concepts in designing polymeric vectors towards meeting therapeutic goals of in vivo and ex vivo gene therapy. Here, we highlight several classes of polymers employed in gene delivery and summarize the recent work on understanding the contributions of chemical and architectural design parameters. We touch upon characterization methods used to visualize and understand events transpiring at the interfaces between polymer, nucleic acids, and the physiological environment. We conclude that interdisciplinary approaches and methodologies motivated by fundamental questions are key to designing high-performing polymeric vehicles for gene therapy.
Bibliographical noteFunding Information:
The authors acknowledge funding support from Limelight Bio (R.K., C.J.G., C.V.B., R.J.D., M.R.B, and J.K.H.), Genentech (M.G.H.), and the National Science Foundation under Award No. DMR-1904853 (C.V.B. and R.J.D.). This work was supported partially by the National Science Foundation through the University of Minnesota Materials Research Science and Engineering Centers (MRSEC) under Award No. DMR-2011401 (C.F.S.C. and M.R.B.). M.G.H. also acknowledges support from the National Science Foundation Graduate Research Fellowship Program (DGE-1839286). Any opinions, findings, and conclusions or recommendations expressed in this material are those of the authors and do not necessarily reflect the views of the National Science Foundation.
PubMed: MeSH publication types
- Journal Article
- Research Support, Non-U.S. Gov't
- Research Support, U.S. Gov't, Non-P.H.S.
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- 2 Active
9/1/20 → 8/31/26
Project: Research project