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

Abstract

Gene therapy is becoming an influential part of the rapidly increasing armamentarium of 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.

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