Gene therapy for the treatment of hemophilia B using PINC-formulated plasmid delivered to muscle with electroporation

Molecular Therapy : the Journal of the American Society of Gene Therapy
J G FewellL C Smith

Abstract

Gene therapy, as a safe and efficacious treatment or prevention of diseases, is one of the next fundamental medical innovations. Direct injection of plasmid into skeletal muscle is still a relatively inefficient and highly variable method of gene transfer. However, published reports have shown that application of an electric field to the muscle immediately after plasmid injection increases gene expression at least 2 orders of magnitude. Using this methodology, we have achieved potentially therapeutic circulating levels of human factor IX (hF.IX) in mice and dogs. A plasmid encoding hF.IX formulated with a protective, interactive, noncondensing (PINC) polymer was injected into the skeletal muscle followed by administration of multiple electrical pulses (electroporation). In mice long-term expression was achieved and the ability to readminister formulated plasmid was demonstrated. In normal dogs, expression of hF.IX reached 0.5-1.0% of normal levels. The transient response in dogs was due to the development of antibodies against hF.IX. Elevated circulating creatine kinase levels and histological examination indicated transient minor trauma associated with the procedure. These data show that gene delivery using a plasmid formulate...Continue Reading

References

Feb 1, 1992·Human Gene Therapy·S JiaoJ A Wolff
Jan 17, 1991·Biochimica Et Biophysica Acta·A V TitomirovE Kistanova
Feb 11, 1997·Biochimica Et Biophysica Acta·M J JaroszeskiR Heller
Apr 7, 1997·Biochemical and Biophysical Research Communications·T MuramatsuJ Okumura
May 27, 1997·Proceedings of the National Academy of Sciences of the United States of America·R W HerzogK A High
Aug 1, 1998·Journal of Controlled Release : Official Journal of the Controlled Release Society·R J MumperA P Rolland
Sep 22, 1998·Nature Biotechnology·H Aihara, J Miyazaki
Jan 8, 1999·Comptes rendus de l'Académie des sciences. Série III, Sciences de la vie·L M MirD Scherman
Apr 14, 1999·Proceedings of the National Academy of Sciences of the United States of America·L M MirD Scherman
May 26, 1999·Proceedings of the National Academy of Sciences of the United States of America·G RizzutoE Fattori
Jul 8, 1999·Human Gene Therapy·R V AbruzzeseJ L Nordstrom
Sep 25, 1999·Human Gene Therapy·R J Kaufman
Oct 9, 1999·Journal of Virology·S E BeckW B Guggino
Jan 5, 2000·Proceedings of the National Academy of Sciences of the United States of America·T GotoY Ushio
Aug 10, 2000·Molecular Therapy : the Journal of the American Society of Gene Therapy·P A FieldsK A High
Sep 14, 2000·Molecular Therapy : the Journal of the American Society of Gene Therapy·S SomiariR W Malone
Sep 14, 2000·Molecular Therapy : the Journal of the American Society of Gene Therapy·M BettanD Scherman
Dec 22, 2000·Molecular Therapy : the Journal of the American Society of Gene Therapy·H ChaoC E Walsh

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Citations

Mar 19, 2002·Advanced Drug Delivery Reviews·Alexander V KabanovValery Alakhov
Dec 14, 2002·International Journal of Pharmaceutics·Indrajit RoySubho Mozumdar
Dec 8, 2005·DNA and Cell Biology·Amir S KhanRuxandra Draghia-Akli
Jun 18, 2010·Human Gene Therapy·Christophe PichavantJacques P Tremblay
Feb 26, 2004·Arthritis Research & Therapy·I-Chuan ChuangLin-Cheng Yang
Mar 27, 2002·Biological & Pharmaceutical Bulletin·Makiya Nishikawa, Mitsuru Hashida
Mar 1, 2011·Radiology and Oncology·Darja PavlinNatasa Tozon
Nov 22, 2005·Expert Opinion on Drug Delivery·Loree C HellerRichard Heller
Mar 29, 2003·Expert Opinion on Biological Therapy·Jason G Fewell, Jeffrey L Nordstrom
Oct 29, 2004·International Journal of Urology : Official Journal of the Japanese Urological Association·Masayuki OtaniShoichi Ueda
Sep 1, 2010·Lower Urinary Tract Symptoms·Masayuki OtaniYukio Homma
Nov 19, 2002·Biochemical and Biophysical Research Communications·Virginie DeleuzeMichel F Bureau
Nov 5, 2005·Journal of Controlled Release : Official Journal of the Controlled Release Society·Jason G FewellKhursheed Anwer
May 24, 2015·General and Comparative Endocrinology·María José MazónAna Gómez
Sep 29, 2004·Molecular Therapy : the Journal of the American Society of Gene Therapy·Patricia A BrownRuxandra Draghia-Akli
Jul 20, 2007·Molecular Therapy : the Journal of the American Society of Gene Therapy·Feng LiuPaul Monahan
Jan 27, 2004·Molecular Therapy : the Journal of the American Society of Gene Therapy·Eric D Rabinovsky, Ruxandra Draghia-Akli
Mar 10, 2004·Molecular Therapy : the Journal of the American Society of Gene Therapy·Hyung-Suk JangDuk-Kyung Kim
Feb 5, 2004·Molecular Therapy : the Journal of the American Society of Gene Therapy·Marco A ArapRenata Pasqualini
Dec 25, 2002·Molecular Therapy : the Journal of the American Society of Gene Therapy·Ruxandra Draghia-AkliRobert H Carpenter
Mar 11, 2003·Technology in Cancer Research & Treatment·Ruxandra Draghia-AkliPatricia A Brown
Apr 23, 2002·The Journal of Histochemistry and Cytochemistry : Official Journal of the Histochemistry Society·Miranda D GroundsMarie A Bogoyevitch
Jan 18, 2008·Technology in Cancer Research & Treatment·D PavlinM Cemazar
Jul 9, 2005·American Journal of Physiology. Cell Physiology·David A Dean
Feb 7, 2003·Gene Therapy·Q L LuT A Partridge
Apr 10, 2004·Cancer Gene Therapy·Catherine M ToneRuxandra Draghia-Akli
May 27, 2004·BioDrugs : Clinical Immunotherapeutics, Biopharmaceuticals and Gene Therapy·Jillian M McMahon, Dominic J Wells
May 11, 2010·Expert Review of Vaccines·Sylvia van Drunen Littel-van den Hurk, D Hannaman
Jun 9, 2019·International Journal of Molecular Sciences·Emilia Sokołowska, Agnieszka Urszula Błachnio-Zabielska
Jan 7, 2003·FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology·Ruxandra Draghia-AkliMarta L Fiorotto
Aug 13, 2002·Current Opinion in Hematology·Paul E Monahan, Gilbert C White
Oct 30, 2004·The Journal of Gene Medicine·Elena FattoriNicola La Monica
Nov 3, 2004·The Journal of Gene Medicine·Yun Chau LongOi Lian Kon

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