Effect of chemical functionalities in poly(amido amine)s for non-viral gene transfection

Research output: Chapter in Book/Report/Conference proceedingConference contributionAcademicpeer-review

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Abstract

The development of safe and efficient gene delivery vectors is an essential prerequisite for successful gene therapy. As viral vectors suffer from inherent disadvantages, cationic polymers as non-viral vectors have great potential in gene delivery, but their practical application so far is seriously hampered due to their relatively low transfection efficiency caused by multiple extra- and intracellular gene delivery barriers. Therefore, it is important to provide cationic polymers with functionalities that can seriously influence polymeric properties which are important to overcome gene delivery barriers. In this paper, we aim to contribute to the understanding of the effect of functionalities in cationic polymers on their gene delivery properties and transfection activity. As poly(amido amine)s can be easily provided with a large variety of chemical functionalities, we have focused on this class of cationic polymers. It is shown that various structural characteristics in these peptidomimetic polymers such as charge density, rigidity, basicity, hydrophilicity/hydrophobicity, degradability and type of amino groups influence one or more gene delivery properties such as DNA binding capability, colloidal stability, endosomal escape (buffer capacity), vector unpacking, cytotoxicity, and eventual transfection efficiency. Optimal combination of the functionalities in the poly(amido amine)s may lead to significant increase of the level of gene expression. This indicates that multifunctionalized polymers like the poly(amido amine)s can evolve to the next generation of non-viral gene delivery system for gene therapy.
Original languageUndefined
Title of host publicationProceedings of the tenth european symposium on controlled drug delivery
EditorsW.E. Hennink, J. Feijen, A.P. Sam
Place of PublicationAmsterdam
PublisherElsevier
Pages267-272
DOIs
Publication statusPublished - 2 Apr 2008
Event10th European Symposium on Controlled Drug Delivery, ESCDD 2008 - Noordwijk aan Zee, Netherlands
Duration: 2 Apr 20084 Apr 2008
Conference number: 10

Publication series

Name3
PublisherElsevier
Number3
Volume132
ISSN (Print)0168-3659
ISSN (Electronic)1873-4995

Conference

Conference10th European Symposium on Controlled Drug Delivery, ESCDD 2008
Abbreviated titleESCDD
CountryNetherlands
CityNoordwijk aan Zee
Period2/04/084/04/08

Keywords

  • METIS-253939
  • IR-69284

Cite this

Lin, C., & Engbersen, J. F. J. (2008). Effect of chemical functionalities in poly(amido amine)s for non-viral gene transfection. In W. E. Hennink, J. Feijen, & A. P. Sam (Eds.), Proceedings of the tenth european symposium on controlled drug delivery (pp. 267-272). (3; Vol. 132, No. 3). Amsterdam: Elsevier. https://doi.org/10.1016/j.jconrel.2008.06.022
Lin, C. ; Engbersen, Johannes F.J. / Effect of chemical functionalities in poly(amido amine)s for non-viral gene transfection. Proceedings of the tenth european symposium on controlled drug delivery. editor / W.E. Hennink ; J. Feijen ; A.P. Sam. Amsterdam : Elsevier, 2008. pp. 267-272 (3; 3).
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title = "Effect of chemical functionalities in poly(amido amine)s for non-viral gene transfection",
abstract = "The development of safe and efficient gene delivery vectors is an essential prerequisite for successful gene therapy. As viral vectors suffer from inherent disadvantages, cationic polymers as non-viral vectors have great potential in gene delivery, but their practical application so far is seriously hampered due to their relatively low transfection efficiency caused by multiple extra- and intracellular gene delivery barriers. Therefore, it is important to provide cationic polymers with functionalities that can seriously influence polymeric properties which are important to overcome gene delivery barriers. In this paper, we aim to contribute to the understanding of the effect of functionalities in cationic polymers on their gene delivery properties and transfection activity. As poly(amido amine)s can be easily provided with a large variety of chemical functionalities, we have focused on this class of cationic polymers. It is shown that various structural characteristics in these peptidomimetic polymers such as charge density, rigidity, basicity, hydrophilicity/hydrophobicity, degradability and type of amino groups influence one or more gene delivery properties such as DNA binding capability, colloidal stability, endosomal escape (buffer capacity), vector unpacking, cytotoxicity, and eventual transfection efficiency. Optimal combination of the functionalities in the poly(amido amine)s may lead to significant increase of the level of gene expression. This indicates that multifunctionalized polymers like the poly(amido amine)s can evolve to the next generation of non-viral gene delivery system for gene therapy.",
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Lin, C & Engbersen, JFJ 2008, Effect of chemical functionalities in poly(amido amine)s for non-viral gene transfection. in WE Hennink, J Feijen & AP Sam (eds), Proceedings of the tenth european symposium on controlled drug delivery. 3, no. 3, vol. 132, Elsevier, Amsterdam, pp. 267-272, 10th European Symposium on Controlled Drug Delivery, ESCDD 2008, Noordwijk aan Zee, Netherlands, 2/04/08. https://doi.org/10.1016/j.jconrel.2008.06.022

Effect of chemical functionalities in poly(amido amine)s for non-viral gene transfection. / Lin, C.; Engbersen, Johannes F.J.

Proceedings of the tenth european symposium on controlled drug delivery. ed. / W.E. Hennink; J. Feijen; A.P. Sam. Amsterdam : Elsevier, 2008. p. 267-272 (3; Vol. 132, No. 3).

Research output: Chapter in Book/Report/Conference proceedingConference contributionAcademicpeer-review

TY - GEN

T1 - Effect of chemical functionalities in poly(amido amine)s for non-viral gene transfection

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AU - Engbersen, Johannes F.J.

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Y1 - 2008/4/2

N2 - The development of safe and efficient gene delivery vectors is an essential prerequisite for successful gene therapy. As viral vectors suffer from inherent disadvantages, cationic polymers as non-viral vectors have great potential in gene delivery, but their practical application so far is seriously hampered due to their relatively low transfection efficiency caused by multiple extra- and intracellular gene delivery barriers. Therefore, it is important to provide cationic polymers with functionalities that can seriously influence polymeric properties which are important to overcome gene delivery barriers. In this paper, we aim to contribute to the understanding of the effect of functionalities in cationic polymers on their gene delivery properties and transfection activity. As poly(amido amine)s can be easily provided with a large variety of chemical functionalities, we have focused on this class of cationic polymers. It is shown that various structural characteristics in these peptidomimetic polymers such as charge density, rigidity, basicity, hydrophilicity/hydrophobicity, degradability and type of amino groups influence one or more gene delivery properties such as DNA binding capability, colloidal stability, endosomal escape (buffer capacity), vector unpacking, cytotoxicity, and eventual transfection efficiency. Optimal combination of the functionalities in the poly(amido amine)s may lead to significant increase of the level of gene expression. This indicates that multifunctionalized polymers like the poly(amido amine)s can evolve to the next generation of non-viral gene delivery system for gene therapy.

AB - The development of safe and efficient gene delivery vectors is an essential prerequisite for successful gene therapy. As viral vectors suffer from inherent disadvantages, cationic polymers as non-viral vectors have great potential in gene delivery, but their practical application so far is seriously hampered due to their relatively low transfection efficiency caused by multiple extra- and intracellular gene delivery barriers. Therefore, it is important to provide cationic polymers with functionalities that can seriously influence polymeric properties which are important to overcome gene delivery barriers. In this paper, we aim to contribute to the understanding of the effect of functionalities in cationic polymers on their gene delivery properties and transfection activity. As poly(amido amine)s can be easily provided with a large variety of chemical functionalities, we have focused on this class of cationic polymers. It is shown that various structural characteristics in these peptidomimetic polymers such as charge density, rigidity, basicity, hydrophilicity/hydrophobicity, degradability and type of amino groups influence one or more gene delivery properties such as DNA binding capability, colloidal stability, endosomal escape (buffer capacity), vector unpacking, cytotoxicity, and eventual transfection efficiency. Optimal combination of the functionalities in the poly(amido amine)s may lead to significant increase of the level of gene expression. This indicates that multifunctionalized polymers like the poly(amido amine)s can evolve to the next generation of non-viral gene delivery system for gene therapy.

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M3 - Conference contribution

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A2 - Hennink, W.E.

A2 - Feijen, J.

A2 - Sam, A.P.

PB - Elsevier

CY - Amsterdam

ER -

Lin C, Engbersen JFJ. Effect of chemical functionalities in poly(amido amine)s for non-viral gene transfection. In Hennink WE, Feijen J, Sam AP, editors, Proceedings of the tenth european symposium on controlled drug delivery. Amsterdam: Elsevier. 2008. p. 267-272. (3; 3). https://doi.org/10.1016/j.jconrel.2008.06.022