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 |
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creator | Foldvari, Marianna Chen, Ding Wen Nafissi, Nafiseh Calderon, Daniella Narsineni, Lokesh Rafiee, Amirreza |
description | 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.
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doi_str_mv | 10.1016/j.jconrel.2015.12.012 |
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subjects | Animals Biodrugs Biopharmaceutics - methods Biopharmaceutics - trends Buccal Cationic lipids Clinical trials Clinical Trials as Topic - methods Cutaneous Delivery of macromolecules Gene editing Gene therapy Gene Transfer Techniques - trends Genetic Therapy - methods Genetic Therapy - trends Humans Intranasal Intravaginal Neoplasms - genetics Neoplasms - metabolism Neoplasms - therapy Non-invasive drug delivery Nucleic acids Ocular Otic Polymers Protein Pulmonary Rectal Transfection Vaccines |
title | Non-viral gene therapy: Gains and challenges of non-invasive administration methods |
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