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
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container_end_page 190
container_issue
container_start_page 165
container_title Journal of controlled release
container_volume 240
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. [Display omitted]
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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