Non-viral gene transfer therapy for cystic fibrosis

Expert Opinion on Biological Therapy
Assem G ZiadyMichael W Konstan

Abstract

Non-viral methods of gene transfer are being investigated to treat cystic fibrosis (CF) and include naked DNA, lipid-DNA complexes and complexes of DNA with polycations such as poly-L-lysine (poly K) or polyethylenimine (PEI), all of which can carry the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The most recent promising strategy is the use of polycation-DNA complexes, particularly those prepared with poly-K and substituted with polyethylene glycol. These complexes produced partial correction of the CF defect in a mouse model with minimal toxicity, and have advanced to clinical trial. Improvements in this and other non-viral methods are in process and include i). targeting the complexes to the desired cells using receptor ligands, ii). lessening toxicity by changing the mix of lipids or adding protective molecules to polycations, iii). modifying the plasmid DNA to reduce inflammatory CpG sequences and enhance intensity, duration and tissue specificity of expression, and iv). modification of the complexes to improve nuclear access.

References

Dec 15, 1991·Proceedings of the National Academy of Sciences of the United States of America·D PapahadjopoulosC Redemann
Aug 1, 1995·Proceedings of the National Academy of Sciences of the United States of America·O BoussifJ P Behr
Aug 11, 1995·The Journal of Biological Chemistry·J ZabnerM J Welsh
Feb 1, 1995·The Journal of Clinical Investigation·T FerkolP B Davis
Dec 1, 1994·European Journal of Biochemistry·J C PeralesR W Hanson
May 31, 1994·Annals of the New York Academy of Sciences·H FarhoodR M Epand
Apr 1, 1994·Human Gene Therapy·J ChenK A Daichendt
Apr 26, 1994·Proceedings of the National Academy of Sciences of the United States of America·J C PeralesR W Hanson
Nov 1, 1993·The Journal of Clinical Investigation·T FerkolP B Davis
Feb 25, 1993·Nucleic Acids Research·P MidouxA C Roche
Mar 15, 1993·Proceedings of the National Academy of Sciences of the United States of America·R J CristianoS L Woo
May 20, 1996·Human Gene Therapy·T SawaJ P Wiener-Kronish
May 1, 1997·Biochemical Society Transactions·C S KaetzelK R Youngman
Jun 1, 1997·American Journal of Respiratory Cell and Molecular Biology·J C DaviesE W Alton
Feb 7, 1998·Proceedings of the National Academy of Sciences of the United States of America·Y H ChowJ Hu
Apr 29, 1998·The Journal of Biological Chemistry·H PollardD Escande
Oct 22, 1998·European Journal of Human Genetics : EJHG·Q Jiang, J F Engelhardt
Jul 30, 1999·Journal of Controlled Release : Official Journal of the Controlled Release Society·W T GodbeyA G Mikos
Jan 15, 2000·The Journal of Biological Chemistry·G L LukacsA S Verkman
Mar 30, 2000·The Journal of Gene Medicine·N ErnstJ Rosenecker
May 10, 2000·Proceedings of the National Academy of Sciences of the United States of America·A C GrothM P Calos
Jul 19, 2000·Molecular Therapy : the Journal of the American Society of Gene Therapy·E AltonM Stern

❮ Previous
Next ❯

Citations

Aug 11, 2011·Molecular Therapy : the Journal of the American Society of Gene Therapy·Jung Soo SukJustin Hanes
Jun 19, 2007·American Journal of Respiratory Cell and Molecular Biology·Benjamin HendricksonCarolyn Lutzko
Sep 1, 2008·Expert Opinion on Drug Discovery·Douglas W Leaman
Apr 26, 2011·Expert Opinion on Biological Therapy·Elahe Mahdipour, Kimberly Ann Mace
Nov 22, 2005·Expert Opinion on Drug Delivery·Stephen Tate, Stuart Elborn
Aug 23, 2007·American Journal of Transplantation : Official Journal of the American Society of Transplantation and the American Society of Transplant Surgeons·H LiuG A Visner
Oct 19, 2004·Biochimica Et Biophysica Acta·Liang WengYan Feng
Oct 7, 2004·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Daniel KlinkBob J Scholte
Jun 21, 2005·Molecular Therapy : the Journal of the American Society of Gene Therapy·John R GreenlandNorman L Letvin
Jan 20, 2011·Journal of Biomaterials Science. Polymer Edition·Wen HeQinmei Wang
Jun 23, 2015·Pediatric Pulmonology·George M SolomonSteven M Rowe

❮ Previous
Next ❯

Related Concepts

Related Feeds

CREs: Gene & Cell Therapy

Gene and cell therapy advances have shown promising outcomes for several diseases. The role of cis-regulatory elements (CREs) is crucial in the design of gene therapy vectors. Here is the latest research on CREs in gene and cell therapy.

Related Papers

Molecular Therapy : the Journal of the American Society of Gene Therapy
Assem-Galal ZiadyPamela B Davis
Molecular Therapy : the Journal of the American Society of Gene Therapy
Assem-Galal ZiadyPamela B Davis
Journal of Biomedical Materials Research. Part B, Applied Biomaterials
Mei LinPiotr Tomasik
Journal of Biomedical Materials Research. Part B, Applied Biomaterials
Hervé NyangogaDaniel Chappard
© 2022 Meta ULC. All rights reserved