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References
Chang, T.M.S. (1997) Artificial cells and bioencapsulation in bioartificial organs. Ann. NY Acad Sci. 831: 249–259.
Huang, J.; Pray, C. and Rozelle, S. (2002) Enhancing the crops to feed the poor. Nature 418(6898): 678–684.
Vajo, Z.; Fawcett, J. and Duckworth, W.C. (2001) Recombinant DNA technology in the treatment of diabetes: insulin analogs. Endocr. Rev. 22(5): 706–717.
Mannucci, P.M. and Giangrande, P.L. (2000) Choice of replacement therapy for hemophilia: recombinant products only? Hematol. J. 1(2): 72–76.
Anderson, W.F.; Blaese, R.M., and Culver, K. (1990) The ADA human gene therapy clinical protocol: Points to Consider response with clinical protocol, July 6, 1990. Hum Gene Ther. 1(3): 331–362.
Teichler Zallen, D. (2000) US gene therapy in crisis. Trends Genet. 16(6): 272–275.
Cavazzana-Calvo, M.; Hacein-Bey, S.; de Saint Basile, G.; Gross, F.; Yvon, E.; Nusbaum, P.; Selz, F.; Hue, C.; Certain, S.; Casanova, J.L.; Bousso, P.; Deist, F.L. and Fischer, A. (2000) Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288(5466): 669–672.
Ledley, F.D. (1995) Hepatic gene therapy. In Chang, P.L. (Ed.) Somatic gene therapy, CRC Press, pp. 61–72.
Lotze, M.T. and Kost, T.A. (2002) Viruses as gene delivery vectors: application to gene function, target validation, and assay development. Cancer Gene Ther. 9(8): 692–699.
Marshall, E. (2001a) Gene therapy. Viral vectors still pack surprises. Science 294: 1640.
Marshall, E. (2001b) Gene therapy. Panel reviews risks of germ line changes. Science 294: 2268–2269.
Lechardeur, D. and Lukacs, G.L. (2002) Intracellular barriers to non-viral gene transfer. Curr Gene Ther 2(2): 183–194.
Matthews, C.; Jenkins, G.; Hilfinger, J. and Davidson, B. (1999) Poly-L-lysine improves gene transfer with adenovirus formulated in PLGA microspheres. Gene Ther. 6(9): 1558–1564.
Sailaja, G.; HogenEsch, H.; North, A.; Hays, J. and Mittal, S.K. (2002) Encapsulation of recombinant adenovirus into alginate microspheres circumvents vector-specific immune response. Gene Ther. 9(24): 1722–1729.
Hortelano, G.; Xu, N.; Vandenberg, A.; Solera, J.; Chang, P.L. and Ofosu, F.A. (1999) Persistent delivery of factor IX in mice: Gene therapy for hemophilia B using implantable microcapsules. Human Gene Ther. 10(8): 1281–1288.
Qin, L.; Ding, Y.; Pahud, D.R.; Chang, E.; Imperiale, M.J. and Bromberg, J.S. (1997) Promoter attenuation in gene therapy: interferon-gamma and tumor necrosis factor-alpha inhibit transgene expression. Hum Gene Ther. 8(17): 2019–2029.
Buchschacher, G.L. Jr. (2001) Introduction to retroviruses and retroviral vectors. Somat. Cell Mol. Genet. 26(1–6): 1–11.
Pannell, D. and Ellis, J. (2001) Silencing of gene expression: implications for design of retrovirus vectors. Rev. Med. Virol. 11(4): 205–217.
Marshall, E. (2002) Gene Therapy a Suspect in Leukemia-like Disease. Science 298: 34–35.
Yee, J.K. and Zaia, J.A. (2001) Prospects for gene therapy using HIV-based vectors. Somat. Cell Mol. Genet. 26(1–6): 159–174.
Breyer, B.; Jiang, W.; Cheng, H.; Zhou, L.; Paul, R.; Feng, T. and He, T.C. (2001) Adenoviral vectormediated gene transfer for human gene therapy. Curr. Gene Ther. 1(2): 149–162.
Trudel, S.; Li, Z., Dodgson, C.; Nanji, S.; Wan, Y.; Voralia, M.; Hitt, M.; Gauldie, J.; Graham, F.L. and Stewart, A.K. (2001) Adenovector engineered interleukin-2 expressing autologous plasma cell vaccination after high-dose chemotherapy for multiple myeloma—a phase 1 study. Leukemia 15(5): 846–854.
DelloRusso, C.; Scott, J.M.; Hartigan-O’Connor, D.; Salvatori, G.; Barjot, C.; Robinson, A.S.; Crawford, R.W.; Brooks, S.V. and Chamberlain J.S. (2002) Functional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin. Proc. Natl. Acad. Sci. USA 99(20): 12979–12984.
Kochanek, S.; Schiedner, G. and Volpers, C. (2001) High-capacity ‘gutless’ adenoviral vectors. Curr. Opin. Mol. Ther. 3(5): 454–463.
Balague, C.; Zhou, J.; Dai, Y.; Alemany, R.; Josephs, S.F.; Andreason, G.; Hariharan, M.; Sethi, E. Prokopenko, E.; Jan, H.Y.; Lou, Y.C.; Hubert-Leslie, D.; Ruiz, L. and Zhang, W.W. (2000) Sustained high-level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector. Blood 95(3): 820–828.
Owens, R.A. (2002) Second generation adeno-associated virus type 2-based gene therapy systems with the potential for preferential integration into AAVS1. Curr. Gene Ther. 2(2): 145–159.
Kay, M.A.; Manno, C.S.; Ragni, M.V.; Larson, P.J.; Couto, L.B.; McClelland, A.; Glader, B.; Chew, A.J.; Tai, S.J.; Herzog, R.W.; Arruda, V.; Johnson, F.; Scallan, C.; Skarsgard, E.; Flake, A.W. and High, K.A. (2000) Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat. Genet. 24(3): 257–261.
Lilley, C.E.; Branston, R.H. and Coffin, R.S. (2001) Herpes simplex virus vectors for the nervous system. Curr. Gene Ther. 1(4): 339–358.
Somiari, S.; Glasspool-Malone, J.; Drabick, J.J.; Gilbert, R.A.; Heller, R.; Jaroszeski, M.J. and Malone, R.W. (2000) Theory and in vivo application of electroporative gene delivery. Mol. Ther. 2(3): 178–187.
Lin, M.T.; Pulkkinen, L.; Uitto, J. and Yoon, K. (2000) The gene gun: current applications in cutaneous gene therapy. Int. J. Dermatol. 39(3): 161–170.
Brauker, J.; Frost, G.H.; Dwarki, V.; Nijjar, T.; Chin, R.; Carr-Brendel, V.; Jasunas, C.; Hodgett, D., Stone, W.; Cohen, L.K.; and Johnson, R.C. (1998) Sustained expression of high levels of human factor IX from human cells implanted within an immunoisolation device into athymic rodents. Hum. Gene Ther. 9(6): 879–888.
Hortelano, G.; Al-Hendy, A.; Ofosu, F.A. and Chang, P.L. (1996) Delivery of human factor IX in mice by microencapsulated recombinant myoblasts: A novel approach towards allogeneic gene therapy of hemophilia B. Blood 87(12): 5095–5103.
Hortelano, G.; Wang, L.; Xu, N. and Ofosu, F.A. (2001) Sustained and therapeutic delivery of human factor IX in nude hemophilia B mice implanted by encapsulated C2C12 myoblasts: concurrent tumorigenesis. Haemophilia 7(2): 207–213.
Liu, H.W.; Ofosu, F.A. and Chang, P.L. (1993) Expression of human factor IX by microencapsulated recombinant fibroblasts. Hum Gene Ther. 4(3): 291–301.
Hughes, M.; Vassilakos, A.; Andrews, D.W.; Hortelano, G.; Belmont, J.W. and Chang, P.L. (1994) Delivery of a secretable adenosine deaminase through microcapsules — a novel approach to somatic gene therapy. Hum Gene Ther. 5(12): 1445–1455.
Al-Hendy, A.; Hortelano, G.; Tannenbaum, G.S. and Chang, P.L. (1995) Correction of the growth defect in dwarf mice with nonautologous microencapsulated myoblasts — an alternate approach to somatic gene therapy. Hum Gene Ther. 6(2): 165–175.
Ross, C.J.; Bastedo, L.; Maier, S.A.; Sands, M.S. and Chang, P.L. (2000) Treatment of a lysosomal storage disease, mucopolysaccharidosis VII, with microencapsulated recombinant cells. Hum Gene Ther. 11(15): 2117–2127.
Chang, P.L.; Van Raamsdonk, J.M.; Hortelano, G.; Barsoum, S.C.; MacDonald, N.C. and Stockley, T.L. (1999) The in vivo delivery of heterologous proteins by microencapsulated recombinant cells. Trends Biotechnol. 17(2): 78–83.
Goto, S.; Miyazaki, K.; Funabiki, T. and Yasumitsu, H. (1999) Serum-free culture conditions for analysis of secretory proteinases during myogenic differentiation of mouse C2C12 myoblasts. Anal. Biochem. 272(2): 135–142.
Gunning, P.; Leavitt, J.; Muscat, G.; Ng, S.Y. and Kedes L. (1987) A human beta-actin expression vector system directs high-level accumulation of antisense transcripts. Proc. Natl. Acad. Sci. USA 84(14): 4831–4835.
Rinsch, C.; Dupraz, P.; Schneider, B.L.; Deglon, N.; Maxwell, P.H.; Ratcliffe, P.J.; Aebischer, P. (2002) Delivery of erythropoietin by encapsulated myoblasts in a genetic model of severe anemia. Kidney Int. 62(4): 1395–1401.
Springer, M.L.; Hortelano, G.; Bouley, D.; Kraft, P.E.; Wong, J. and Blau, H.M. (2000) Induction of angiogenesis by implantation of encapsulated primary myoblasts expressing vascular endothelial growth factor. J. Gene Med. 2(4): 279–288.
Rinsch, C.; Peduto, G.; Schneider, B.L. and Aebischer, P. (2001) Inducing host acceptance to encapsulated xenogeneic myoblasts. Transplantation 71(3): 345–351.
Aebischer, P.; Schluep, M.; Deglon, N.; Joseph, J.M.; Hirt, L.; Heyd, B.; Goddard, M.; Hammang, J.P.; Zurn, A.D.; Kato, A.C.; Regli, F. and Baetge, E.E. (1996) Intrathecal delivery of CNTF using encapsulated genetically modified xenogeneic cells in amyotrophic lateral sclerosis patients. Nat. Med. 2(6): 696–699.
Buchser, E.; Goddard, M.; Heyd, B.; Joseph, J.M.; Favre, J.; de Tribolet, N.; Lysaght, M. and Aebischer, P. (1996) Immunoisolated xenogenic chromaffin cell therapy for chronic pain. Initial clinical experience. Anesthesiology 85(5): 1005–1012.
Piskin, E. (2002). Biodegradable polymeric matrices for bioartificial implants. Int. J. Artif. Organs 25(5): 434–440.
Cirone, P.; Bourgeois, J.M.; Austin, R.C. and Chang, P.L. (2002). A novel approach to tumor suppression with microencapsulated recombinant cells. Hum. Gene Ther. 13(10): 1157–1166.
Read, T.A.; Sorensen, D.R.; Mahesparan, R.; Enger, P.O.; Timpl, R.; Olsen, B.R.; Hjelstuen, M.H.; Haraldseth, O. and Bjerkvig, R. (2001) Local endostatin treatment of gliomas administered by microencapsulated producer cells. Nat. Biotechnol. 19(1): 29–34.
Muller, P.; Jesnowski, R.; Karle, P.; Renz, R.; Saller, R.; Stein, H.; Puschel, K.; von Rombs, K.; Nizze, H.; Liebe, S.; Wagner, T.; Gunzburg, W.H.; Salmons, B. and Lohr, M. (1999) Injection of encapsulated cells producing an ifosfamide-activating cytochrome P450 for targeted chemotherapy to pancreatic tumors. Ann. NY Acad. Sci. 880: 337–351.
Lohr, M.; Hummel, F.; Faulmann, G.; Ringel, J.; Saller, R.; Hain, J.; Gunzburg, W.H. and Salmons, B. (2002) Microencapsulated, CYP2B1-transfected cells activating ifosfamide at the site of the tumor: the magic bullets of the 21st century. Cancer Chemother. Pharmacol. 49Suppl. 1: S21–24.
Lohr, M.; Hoffmeyer, A.; Kroger, J.; Freund, M.; Hain, J.; Holle, A.; Karle, P.; Knofel, W.T.; Liebe, S.; Muller, P.; Nizze, H.; Renner, M.; Saller, R.M.; Wagner, T.; Hauenstein, K.; Gunzburg, W.H. and Salmons, B. (2001) Microencapsulated cell-mediated treatment of inoperable pancreatic carcinoma. Lancet 357(9268): 1591–1592.
Geller, R.L.; Neuenfeldt, S.; Levon, S.A.; Maryanov, D.A.; Thomas, T.J. and Brauker, J.H. (1997) Immunoisolation of tumor cells: generation of antitumor immunity through indirect presentation of antigen. J. Immunother. 20(2): 131–137.
Huber, A.; Padrun, V.; Deglon, N.; Aebischer, P.; Mohler, H. and Boison, D. (2001) Grafts of adenosinereleasing cells suppress seizures in kindling epilepsy. Proc. Natl. Acad. Sci. USA 98(13): 7611–7616.
Deglon, N.; Heyd, B.; Tan, S.A.; Joseph, J.M.; Zurn, A.D. and Aebischer, P. (1996) Central nervous system delivery of recombinant ciliary neurotrophic factor by polymer encapsulated differentiated C2C12 myoblasts. Hum. Gene Ther. 7(17): 2135–2146.
Zurn, A.D.; Widmer, H.R. and Aebischer, P. (2001) Sustained delivery of GDNF: towards a treatment for Parkinson’s disease. Brain Res. Rev. 36(2–3): 222–229.
Zurn, A.D.; Henry, H.; Schluep, M.; Aubert, V.; Winkel, L.; Eilers, B.; Bachmann, C. and Aebischer, P. (2000) Evaluation of an intrathecal immune response in amyotrophic lateral sclerosis patients implanted with encapsulated genetically engineered xenogeneic cells. Cell Transplant. 9(4): 471–484.
Emerich, D.F.; Cain, C.K.; Greco, C.; Saydoff, J.A.; Hu, Z.Y.; Liu, H. and Lindner, M.D. (1997) Cellular delivery of human CNTF prevents motor and cognitive dysfunction in a rodent model of Huntington’s disease. Cell Transplant. 6(3): 249–266.
Saitoh, Y.; Eguchi, Y.; Hagihara, Y.; Arita, N.; Watahiki, M.; Tsujimoto, Y. and Hayakawa, T. (1998) Dose-dependent doxycycline-mediated adrenocorticotropic hormone secretion from encapsulated Tet-on proopiomelanocortin Neuro2A cells in the subarachnoid space. Hum. Gene Ther. 9(7): 997–1002.
Sommer, B.; Rinsch, C.; Payen, E.; Dalle, B.; Schneider, B.; Deglon, N.; Henri, A.; Beuzard, Y. and Aebischer, P. (2002). Long-term doxycycline-regulated secretion of erythropoietin by encapsulated myoblasts. Mol. Ther. 6(2): 155–161.
Dalle, B.; Payen, E.; Regulier, E.; Deglon, N.; Rouyer-Fessard, P.; Beuzard, Y. and Aebischer, P. (1999) Improvement of mouse beta-thalassemia upon erythropoietin delivery by encapsulated myoblasts. Gene Ther. 6(2): 157–161.
Hedner, U. and Davie, E.W. (1989) Introduction to hemostasis and the vitamin K-dependent coagulation factors. In Scriver, C.R.; Beaudet, A.L.; Sly, W.S. and Valle, D. (Eds.) The metabolic basis of inherited disease, vol. II, 6th edition, McGraw-Hill, New York; pp. 2107.
Gianelli, F; Green, P.M.; High, K.A.; Sommer, S.; Poon, M.C.; Ludwig, M., Schwaab, R.; Reitsma, P.H.; Goossens, M. and Yoshioka, A. (1993) Haemophilia B: database of point mutations and short additions and deletions-third edition. Nucleic Acids Res. 21: 3075–3087.
Roberts, H.R. and Eberst, M.E. (1993) Current management of hemophilia B. Hematology/Oncology clinics of North America 7(6): 1269–1279.
Walker, I. (1997) Recombinant factor IX has been licensed for use. Hemophilia Today (Summer).
Kay, M.A.; Rothenberg, S.; Landen, C.N.; Bellinger, D.A.; Leland, F.; Toman, C.; Finegold, M.; Thompson, A.R.; Read, M.S.; Brinkhous, K.M.; et al. (1993). In vivo gene therapy for hemophilia B: Sustained partial correction in factor IX deficient dogs. Science 262: 117–119.
Bi, L.; Lawler, A.M.; Antonarakis, S.E.; High, KA; Gearhart, JD and Kazazian, HH Jr. (1995). Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A. Nat Genet 10(1):119–121
Lin, H.F.; Maeda, N.; Smithies, O.; Straight, D.L. and Stafford, D.W. (1997) A coagulation factor IXdeficient mouse model for human hemophilia B. Blood 90(10): 3962–3966.
Giles, A.R.; Tinlin, S. and Greenwood, R. (1982) A canine model of hemophilic (factor VIII:C deficiency) bleeding. Blood 60(3): 727–730.
Evans, J.P.; Brinkhous, K.M.; Brayer, G.D.; Reisner, H. and High, K.A. (1989) Canine hemophilia B resulting from a point mutation with unusual consequences. Proc. Natl. Acad. Sci. USA 86(24): 10095–10099.
Wang, L.; Zoppe, M.; Hackeng, T.M.; Griffin, J.H.; Lee, K.F. and Verma, I.M. (1997) A factor IXdeficient mouse model for hemophilia B gene therapy. Proc. Natl. Acad. Sci. USA 94(21): 11563–11566.
Kay, M. and High, K. (1999) Gene therapy for the hemophilias. Proc. Natl. Acad. Sci. USA 96: 9973–9975.
Wang, L.; Takabe, K.; Bidlingmaier, S.M.; Ill, C.R. and Verma, I.M. (1999) Sustained correction of bleeding disorder in hemophilia B mice by gene therapy. Proc. Natl. Acad. Sci. USA 96(7): 3906–3910.
Garcia-Martin, C.; Chuah, M.K.; Van Damme, A.; Robinson, K.E.; Vanzieleghem, B.; Saint-Remy, J.M.; Gallardo, D.; Ofosu, F.A.; Vandendriessche, T. and Hortelano, G. (2002) Therapeutic levels of human factor VIII in mice implanted with encapsulated cells: potential for gene therapy of haemophilia A. J. Gene Med. 4(2): 215–223.
Saller, R.M.; Indraccolo, S.; Coppola, V.; Esposito, G.; Stange, J.; Mitzner, S.; Amadori, A.; Salmons, B. and Gunzburg, W.H. (2002) Encapsulated cells producing retroviral vectors for in vivo gene transfer. J. Gene Med. 4(2): 150–160.
Prakash, S. and Chang, T.M. (1996). Microencapsulated genetically engineered live E. coli DH5 cells administered orally to maintain normal plasma urea level in uremic rats. Nat. Med. 2(8): 883–887.
Birney, E.; Bateman, A.; Clamp, M.E. and Hubbard, T.J. (2001) Mining the draft human genome. Nature 409: 827–828.
Dautzenberg, H.; Schuldt, U.; Grasnick, G.; Karle, P.; Muller, P.; Lohr, M.; Pelegrin, M.; Piechaczyk, M.; Rombs, K.V.; Gunzburg, W.H.; Salmons, B. and Saller, R.M. (1999) Development of cellulose sulfatebased polyelectrolyte complex microcapsules for medical applications. Ann. NY Acad. Sci. 875: 46–63.
Lahooti, S. and Sefton, M.V. (2000) Effect of an immobilization matrix and capsule membrane permeability on the viability of encapsulated HEK cells. Biomaterials 21(10): 987–995.
Joki, T.; Machluf, M.; Atala, A.; Zhu, J.; Seyfried, N.T.; Dunn, I.F.; Abe, T.; Carroll, R.S. and Black, P.M. (2001) Continuous release of endostatin from microencapsulated engineered cells for tumor therapy. Nat. Biotechnol. 19(1): 35–39.
Weber, M.; Steinert, A.; Jork, A.; Dimmler, A.; Thurmer, F.; Schutze, N.; Hendrich, C. and Zimmerman, U. (2002) Formation of cartilage matrix proteins by BMP-transfected murine mesenchymal stem cells encapsulated in a novel class of alginates. Biomaterials 23(9): 2003–2013.
Rokstad, A.M.; Holtan, S.; Strand, B.; Steinkjer, B.; Ryan, L.; Kulseng, B.; Skjak-Braek, G. and Espevik, T. (2002) Microencapsulation of cells producing therapeutic proteins: optimizing cell growth and secretion. Cell Transplant. 11(4): 313–324.
Naganawa, Y.; Ohsugi, K.; Kase, R.; Date, I.; Sakuraba, H. and Sakuragawa, N. (2002) In vitro study of encapsulation therapy for Fabry disease using genetically engineered CHO cell line. Cell Transplant. 11(4): 325–329.
Date, I.; Shingo, T.; Yoshida, H.; Fujiwara, K.; Kobayashi, K. and Ohmoto, T. (2000) Grafting of encapsulated dopamine-secreting cells in Parkinson’s disease: long-term primate study. Cell Transplant. 9(5): 705–709.
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Hortelano, G. (2005). Gene Therapy Using Encapsulated Cells. In: Nedović, V., Willaert, R. (eds) Applications of Cell Immobilisation Biotechnology. Focus on Biotechnology, vol 8B. Springer, Dordrecht. https://doi.org/10.1007/1-4020-3363-X_13
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