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Poly(beta-amino ester)s as gene delivery vehicles: challenges and opportunities
Department of Biomedical Engineering and the Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD, USA.ORCID iD: 0000-0001-6008-6692
Department of Biomedical Engineering and the Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Department of Biomedical Engineering and the Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD, USA; Johns Hopkins University, Baltimore, MD, USA.
Department of Biomedical Engineering and the Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
2020 (English)In: Expert Opinion on Drug Delivery, ISSN 1742-5247, E-ISSN 1744-7593, Vol. 17, no 10, p. 1395-1410Article in journal (Refereed) Published
Abstract [en]

INTRODUCTION: Gene delivery technologies are being developed for an increasing number of biomedical applications, with delivery vehicles including viruses and non-viral materials. Among biomaterials used for non-viral gene delivery, poly(beta-amino ester)s (PBAEs), a class of synthetic, biodegradable polymers, have risen as a leading gene delivery vehicle that has been used for multiple applications in vitro and in vivo.

AREAS COVERED: This review summarizes the key properties of PBAEs and their development, including a discussion of the advantages and disadvantages of PBAEs for gene delivery applications. The use of PBAEs to improve the properties of other drug delivery vehicles is also summarized.

EXPERT OPINION: PBAEs are designed to have multiple characteristics that are ideal for gene delivery, including their reversible positive charge, which promotes binding to nucleic acids as well as imparting high buffering capacity, and their rapid degradability under mild conditions. Simultaneously, some of their properties also lead to nanoparticle instability and low transfection efficiency in physiological environments. The ease with which PBAEs can be chemically modified as well as non-covalently blended with other materials, however, allows them to be customized specifically to overcome delivery barriers for varied applications.

Place, publisher, year, edition, pages
Taylor & Francis, 2020. Vol. 17, no 10, p. 1395-1410
Keywords [en]
Biomaterials, gene delivery, nanoparticles, nucleic acids
National Category
Polymer Chemistry
Identifiers
URN: urn:nbn:se:liu:diva-205986DOI: 10.1080/17425247.2020.1796628PubMedID: 32700581OAI: oai:DiVA.org:liu-205986DiVA, id: diva2:1885097
Available from: 2024-07-22 Created: 2024-07-22 Last updated: 2024-10-30Bibliographically approved

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Karlsson, Johan

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