Delivery of therapeutic oligonucleotides with cell penetrating peptides.

Advanced Drug Delivery Reviews
Prisca BoisguerinBernard Lebleu

Abstract

Oligonucleotide-based drugs have received considerable attention for their capacity to modulate gene expression very specifically and as a consequence they have found applications in the treatment of many human acquired or genetic diseases. Clinical translation has been often hampered by poor biodistribution, however. Cell-penetrating peptides (CPPs) appear as a possibility to increase the cellular delivery of non-permeant biomolecules such as nucleic acids. This review focuses on CPP-delivery of several classes of oligonucleotides (ONs), namely antisense oligonucleotides, splice switching oligonucleotides (SSOs) and siRNAs. Two main strategies have been used to transport ONs with CPPs: covalent conjugation (which is more appropriate for charge-neutral ON analogues) and non-covalent complexation (which has been used for siRNA delivery essentially). Chemical synthesis, mechanisms of cellular internalization and various applications will be reviewed. A comprehensive coverage of the enormous amount of published data was not possible. Instead, emphasis has been put on strategies that have proven to be effective in animal models of important human diseases and on examples taken from the authors' own expertise.

References

Jan 1, 1978·Proceedings of the National Academy of Sciences of the United States of America·P C Zamecnik, M L Stephenson
Jan 1, 1978·Proceedings of the National Academy of Sciences of the United States of America·M L Stephenson, P C Zamecnik
Aug 1, 1978·Proceedings of the National Academy of Sciences of the United States of America·H J Ryser, W C Shen
Feb 1, 1984·Proceedings of the National Academy of Sciences of the United States of America·G BulfieldK J Moore
Mar 1, 1995·The Journal of Cell Biology·B AllinquantA Prochiantz
Jul 15, 1997·Nucleic Acids Research·M C MorrisG Divita
Apr 16, 1999·Muscle & Nerve·T I Samoylova, B F Smith
Aug 14, 1999·Nucleic Acids Research·M C MorrisG Divita
Dec 29, 1999·Human Mutation·A HamoshV A McKusick
Nov 23, 2000·Proceedings of the National Academy of Sciences of the United States of America·P A WenderJ B Rothbard
Nov 30, 2000·The Journal of Peptide Research : Official Journal of the American Peptide Society·D J MitchellJ B Rothbard
Sep 27, 2002·Current Opinion in Pharmacology·Mark A Lindsay
Nov 12, 2002·Nature Biotechnology·Peter SazaniRyszard Kole
Mar 3, 2004·Biochemistry·Stephen M Fuchs, Ronald T Raines
Mar 18, 2004·Bioconjugate Chemistry·Hong M MoultonPatrick L Iversen
Aug 7, 2004·European Journal of Pharmaceutics and Biopharmaceutics : Official Journal of Arbeitsgemeinschaft Für Pharmazeutische Verfahrenstechnik E.V·Rachel Tréhin, Hans P Merkle
Nov 27, 2004·Molecular Therapy : the Journal of the American Society of Gene Therapy·Ikuhiko NakaseShiroh Futaki
Dec 8, 2004·Biochimica Et Biophysica Acta·Sébastien DeshayesFrédéric Heitz
Jun 22, 2005·Cellular and Molecular Life Sciences : CMLS·S DeshayesF Heitz
Oct 11, 2005·Biochimica Et Biophysica Acta·Mazin MagzoubAstrid Gräslund
Oct 29, 2005·Chembiochem : a European Journal of Chemical Biology·Rainer FischerRoland Brock
Dec 27, 2005·Journal of Controlled Release : Official Journal of the Controlled Release Society·Saïd AbesBernard Lebleu
Mar 17, 2006·Biochimica Et Biophysica Acta·Antoine KichlerBurkhard Bechinger
Jun 13, 2006·Molecular Therapy : the Journal of the American Society of Gene Therapy·Won Jong KimSung Wan Kim
Aug 11, 2006·The Journal of Gene Medicine·Samir El-AndaloussiUlo Langel
Sep 15, 2006·Nature Reviews. Molecular Cell Biology·Kay E Davies, Kristen J Nowak
Nov 14, 2006·Journal of Controlled Release : Official Journal of the Controlled Release Society·Saïd AbesBernard Lebleu

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Citations

Feb 16, 2016·Carbohydrate Polymers·Altanzul AltangerelJingfen Han
Jan 14, 2016·Chemical Society Reviews·Juan LiWeihong Tan
Oct 20, 2015·Journal of Controlled Release : Official Journal of the Controlled Release Society·Y SuzukiH Ishihara
Aug 19, 2015·Current Opinion in Pharmacology·Graham McClorey, Matthew J Wood
Mar 12, 2016·ACS Applied Materials & Interfaces·Hai XiaoHuricha Baigude
Mar 29, 2016·Bioconjugate Chemistry·Yoshimasa KawaguchiShiroh Futaki
Apr 17, 2016·Nucleic Acids Research·Rudolph L Juliano
May 26, 2016·International Journal of Pharmaceutics·Karidia KonatePrisca Boisguerin
Jun 9, 2016·Journal of Controlled Release : Official Journal of the Controlled Release Society·Egon UrgardAna Rebane
Jun 19, 2016·Biochimie·Elena M SmekalovaVictor Koteliansky
Oct 31, 2016·Advanced Drug Delivery Reviews·Taavi LehtoSamir El Andaloussi
Sep 7, 2016·Trends in Biotechnology·Christian Rüter, M Alexander Schmidt
Sep 13, 2016·ACS Applied Materials & Interfaces·Yuhuan LiLesheng Teng
Oct 12, 2016·Chemical & Pharmaceutical Bulletin·Toshihide Takeuchi, Shiroh Futaki
Oct 21, 2016·Bioconjugate Chemistry·Martina JezowskaMalgorzata Honcharenko
Dec 5, 2016·Nucleic Acids Research·Trish T Hoang, Ronald T Raines
Apr 12, 2017·Wiley Interdisciplinary Reviews. Nanomedicine and Nanobiotechnology·Michael E NanceDongsheng Duan
Apr 6, 2017·Molecules : a Journal of Synthetic Chemistry and Natural Product Chemistry·Valentina SardoneMaria Sofia Falzarano
May 23, 2017·Natural Product Reports·P Klahn, M Brönstrup
Jun 21, 2017·International Journal of Pharmaceutics·Xi-Rui ZhouShi-Zhong Luo
Jun 15, 2017·Chembiochem : a European Journal of Chemical Biology·Maria TaskovaKira Astakhova
Dec 23, 2015·International Journal of Molecular Sciences·Buddhadev LayekJagdish Singh
Oct 14, 2017·Molecules : a Journal of Synthetic Chemistry and Natural Product Chemistry·Yating SunChenguang Zhou
Jan 10, 2018·Medicinal Research Reviews·Changmai ChenXinjing Tang
May 8, 2018·Biomedicines·Graham McClorey, Subhashis Banerjee
May 5, 2018·Expert Opinion on Drug Delivery·Kevin CraigMansoor Amiji
Aug 8, 2015·Angewandte Chemie·Céline DouatGilles Guichard
Oct 17, 2018·Chembiochem : a European Journal of Chemical Biology·Jasmine ChamioloOliver Seitz
Jan 27, 2019·Chembiochem : a European Journal of Chemical Biology·Hao JiangCarsten Schmuck
Mar 16, 2017·EMBO Molecular Medicine·Caroline GodfreyVirginia Arechavala-Gomeza
Apr 19, 2019·Chembiochem : a European Journal of Chemical Biology·Makoto Oba
Nov 7, 2019·Journal of Peptide Science : an Official Publication of the European Peptide Society·Kun JiWei Zhang

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Methods Mentioned

BETA
chemical modifications
chemical modification
PCR
phage display
genetic modification
SMA
circular dichroism
atomic force microscopy
transfection
light scattering

Software Mentioned

Transportan

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