4.8 Article

Non-viral gene therapy: Gains and challenges of non-invasive administration methods

Journal

JOURNAL OF CONTROLLED RELEASE
Volume 240, Issue -, Pages 165-190

Publisher

ELSEVIER SCIENCE BV
DOI: 10.1016/j.jconrel.2015.12.012

Keywords

Non-invasive drug delivery; Delivery of macromolecules; Gene therapy; Biodrugs; Protein; Nucleic acids; Vaccines; Transfection; Cutaneous; Ocular; Pulmonary; Buccal; Intranasal; Intravaginal; Rectal; Otic; Clinical trials; Gene editing; Cationic lipids; Polymers

Funding

  1. Canadian Institutes of Health Research
  2. Natural Sciences and Engineering Research Council of Canada
  3. Canada Foundation for Innovation
  4. Ontario Research Fund
  5. Canada Research Chairs Program the Canada Foundation for Innovation

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Gene therapy is becoming an influential part of the rapidly increasing armamentariumof biopharmaceuticals for improving health and combating diseases. Currently, three gene therapy treatments are approved by regulatory agencies. While these treatments utilize viral vectors, non-viral alternative technologies are also being developed to improve the safety profile and manufacturability of gene carrier formulations. We present an overview of gene-based therapies focusing on non-viral gene delivery systems and the genetic therapeutic tools that will further revolutionize medical treatment with primary focus on the range and development of non-invasive delivery systems for dermal, transdermal, ocular and pulmonary administrations and perspectives on other administration methods such as intranasal, oral, buccal, vaginal, rectal and otic delivery. (C) 2015 Elsevier B.V. All rights reserved.

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