PMID: 11933222Apr 5, 2002Paper

Therapeutic levels of human factor VIII in mice implanted with encapsulated cells: potential for gene therapy of haemophilia A

The Journal of Gene Medicine
Carmen García-MartínGonzalo Hortelano

Abstract

A gene therapy delivery system based on microcapsules enclosing recombinant cells engineered to secrete a therapeutic protein has been evaluated. The microcapsules are implanted intraperitoneally. In order to prevent cell immune rejection, cells are enclosed in non-antigenic biocompatible alginate microcapsules prior to their implantation into mice. It has been shown that encapsulated myoblasts can deliver therapeutic levels of Factor IX (FIX) in mice. The delivery of human Factor VIII (hFVIII) in mice using microcapsules was evaluated in this study. Mouse C2C12 myoblasts and canine MDCK epithelial kidney cells were transduced with MFG-FVIII (B-domain deleted) vector. Selected recombinant clones were enclosed in alginate microcapsules. Encapsulated recombinant clones were subsequently implanted intraperitoneally into C57BL/6 and immunodeficient SCID mice. Plasma of mice receiving C2C12 and encapsulated MDCK cells had transient therapeutic levels of FVIII in immunocompetent C57BL/6 mice (up to 20% and 7% of physiological levels, respectively). In addition, FVIII delivery in SCID mice was also transient, suggesting that a non-immune mechanism must have contributed to the decline of hFVIII in plasma. Quantitative RT-PCR analysis c...Continue Reading

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Citations

Jul 11, 2006·Biotechnology Letters·Yeming WangXiaolin Li
Nov 4, 2008·Biotechnology Letters·Shen Youjin, Yin Jun
Feb 1, 2003·Haemophilia : the Official Journal of the World Federation of Hemophilia·A Van DammeT VandenDriessche
Oct 28, 2003·Human Gene Therapy·Therese VistedMorten Lund-Johansen
Sep 23, 2006·Expert Opinion on Biological Therapy·Shu Uin GanRoy Y Calne
Aug 15, 2009·Journal of Cellular Biochemistry·Daniel L CoutuJacques Galipeau
Jan 10, 2012·Haemophilia : the Official Journal of the World Federation of Hemophilia·Q ShiR R Montgomery
Jun 30, 2010·Journal of Biomedical Materials Research. Part B, Applied Biomaterials·Ajit ThakurGonzalo Hortelano
Apr 14, 2016·Molecular Therapy. Methods & Clinical Development·Graça Almeida-PoradaChristopher D Porada
Jun 17, 2006·Neurobiology of Disease·Dwaine F EmerichJeffrey H Kordower
Jul 2, 2003·Expert Opinion on Biological Therapy·Therese Visted, Morten Lund-Johansen
Jan 8, 2015·Frontiers in Pharmacology·Christopher D PoradaGraça Almeida-Porada
Oct 11, 2003·Arteriosclerosis, Thrombosis, and Vascular Biology·Christian HerderErhard Seifried
Nov 8, 2006·Journal of Biomedical Materials Research. Part a·C G ThanosD F Emerich
Dec 19, 2019·Frontiers in Bioengineering and Biotechnology·Assem AshimovaGonzalo Hortelano

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