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

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 te...

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Veröffentlicht in:Journal of controlled release 2016-10, Vol.240, p.165-190
Hauptverfasser: Foldvari, Marianna, Chen, Ding Wen, Nafissi, Nafiseh, Calderon, Daniella, Narsineni, Lokesh, Rafiee, Amirreza
Format: Artikel
Sprache:eng
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Zusammenfassung: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. [Display omitted]
ISSN:0168-3659
1873-4995
DOI:10.1016/j.jconrel.2015.12.012