Designer peptide delivery systems for gene therapy

Stephen Patrick Loughran, Cian Michael McCrudden, Helen Olga McCarthy

Research output: Contribution to journalReview article

9 Citations (Scopus)
269 Downloads (Pure)

Abstract

Gene therapy has long been hailed as a revo- lutionary approach for the treatment of genetic diseases. The enthusiasm that greeted the harnessing of viruses for therapeutic DNA delivery has been tempered by concerns over safety. These concerns led to the development of alternative strategies for nucleic acid delivery to cells. One such strategy is the utilization of cationic peptides for the condensation of therapeutic DNA for delivery to its target. However, success of DNA as a therapy relies on its delivery to the nucleus of target cells, a process that is complicated by the many hurdles encountered following systemic administration. Non-viral peptide gene delivery strategies have sought inspiration from viruses in order to retain DNA delivering potency, but limit virulence. This review summarizes the progression of peptide-based DNA deliv- ery systems, from rudimentary beginnings to the recent development of sophisticated multi-functional vectors that comprise distinct motifs with dedicated barrier eva- sion functions. The most promising peptides that achieve cell membrane permeabilization, endosomal escape and nuclear delivery are discussed.
Original languageEnglish
Pages (from-to)85-96
Number of pages12
JournalEuropean Journal of Nanomedicine
Volume7
Issue number2
Early online date21 Mar 2015
DOIs
Publication statusPublished - Apr 2015

Fingerprint

gene therapy
Gene therapy
Genetic Therapy
Peptides
peptides
delivery
DNA
deoxyribonucleic acid
Viruses
viruses
Inborn Genetic Diseases
DNA Viruses
Therapeutics
Cell Nucleus
Nucleic Acids
Nucleic acids
Virulence
Cell membranes
virulence
inspiration

Bibliographical note

Specialist Publication following the Translational Nanomedicine Conference in 2014

Cite this

Loughran, Stephen Patrick ; McCrudden, Cian Michael ; McCarthy, Helen Olga. / Designer peptide delivery systems for gene therapy. In: European Journal of Nanomedicine. 2015 ; Vol. 7, No. 2. pp. 85-96.
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Designer peptide delivery systems for gene therapy. / Loughran, Stephen Patrick; McCrudden, Cian Michael; McCarthy, Helen Olga.

In: European Journal of Nanomedicine, Vol. 7, No. 2, 04.2015, p. 85-96.

Research output: Contribution to journalReview article

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AB - Gene therapy has long been hailed as a revo- lutionary approach for the treatment of genetic diseases. The enthusiasm that greeted the harnessing of viruses for therapeutic DNA delivery has been tempered by concerns over safety. These concerns led to the development of alternative strategies for nucleic acid delivery to cells. One such strategy is the utilization of cationic peptides for the condensation of therapeutic DNA for delivery to its target. However, success of DNA as a therapy relies on its delivery to the nucleus of target cells, a process that is complicated by the many hurdles encountered following systemic administration. Non-viral peptide gene delivery strategies have sought inspiration from viruses in order to retain DNA delivering potency, but limit virulence. This review summarizes the progression of peptide-based DNA deliv- ery systems, from rudimentary beginnings to the recent development of sophisticated multi-functional vectors that comprise distinct motifs with dedicated barrier eva- sion functions. The most promising peptides that achieve cell membrane permeabilization, endosomal escape and nuclear delivery are discussed.

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