Development of engineered cells for implantation in gene therapy

Advanced Drug Delivery Reviews - Tập 33 - Trang 31-43 - 1998
Patricia L. Chang1,2,3, Kelly MacMillan Bowie1
1Department of Biology, McMaster University, Hamilton, Ontario, Canada
2Department of Pediatrics, McMaster University, Hamilton, Ontario, Canada
3Department of Biomedical Sciences, McMaster University, Hamilton, Ontario, Canada

Tài liệu tham khảo

Ross, 1996, Gene therapy in the United States: a five year status report, Hum. Gene Ther., 7, 1781, 10.1089/hum.1996.7.14-1781 Blau, 1995, Gene therapy – A novel form of drug delivery, N. Engl. J. Med., 333, 1204, 10.1056/NEJM199511023331808 P.L. Chang, Nonautologous somatic gene therapy, in: P.L. Chang (Ed.), Somatic Gene Therapy, CRC Press, Boca Raton, FL, 1995, pp. 203–223. Stockley, 1997, Nonautologous somatic gene therapy: a novel approach to drug delivery, Drug News Perspect., 10, 331 J.M. Rappeport, Indications and applications of bone marrow transplantation, in: R. Hoffman, E.J. Benz Jr., S.J. Shattil, B. Furie, H.J. Cohen (Eds.), Hematology: Basic Principles and Practice, Churchill Livingstone, New York, NY, 1991, pp. 230–242. Kohn, 1995, Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency, Nat. Med., 1, 1017, 10.1038/nm1095-1017 Blaese, 1995, T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4 years, Science, 270, 475, 10.1126/science.270.5235.475 Bordignon, 1995, Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients, Science, 270, 470, 10.1126/science.270.5235.470 Seldon, 1987, Implantation of genetically engineered fibroblasts into mice: implications for gene therapy, Science, 236, 714, 10.1126/science.3472348 Chang, 1990, Autologous fibroblast implantation: feasibility and potential problems in gene replacement therapy, Mol. Biol. Med., 7, 461 Seppen, 1997, Transplantation of Gunn rats with autologous fibroblasts expressing bilirubin UDP-glucuronosyltransferase: correction of genetic deficiency and tumor formation, Hum. Gene Ther., 8, 27, 10.1089/hum.1997.8.1-27 Grossman, 1994, Successful ex vivo gene therapy directed to liver in a patient with familial hypercholesterolaemia, Nat. Genet., 6, 335, 10.1038/ng0494-335 Snyder, 1997, Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors, Nat. Genet., 16, 270, 10.1038/ng0797-270 Choate, 1996, Corrective gene transfer in the human skin disorder lamellar ichthyosis, Nat. Med., 2, 1263, 10.1038/nm1196-1263 Fenjves, 1994, Systemic delivery of secreted protein by grafts of epidermal keratinocytes: prospects for keratinocyte gene therapy, Hum. Gene Ther., 5, 1241, 10.1089/hum.1994.5.10-1241 Gerrard, 1993, Towards gene therapy for haemophilia B using primary human keratinocytes, Nat. Genet., 3, 180, 10.1038/ng0293-180 Barr, 1991, Systemic delivery of recombinant proteins by genetically modified myoblasts, Science, 254, 1507, 10.1126/science.1962212 Yao, 1992, Expression of human factor IX in mice after injection of genetically modified myoblasts, Proc. Natl. Acad. Sci. USA, 89, 3357, 10.1073/pnas.89.8.3357 Naffakh, 1996, Long-term secretion of therapeutic proteins from genetically modified skeletal muscles, Hum. Gene Ther., 7, 11, 10.1089/hum.1996.7.1-11 Shull, 1996, Myoblast gene therapy in canine mucopolysaccharidosis I: abrogation by an immune response to L-iduronidase, Hum. Gene Ther., 7, 1595, 10.1089/hum.1996.7.13-1595 Herzog, 1997, Stable gene transfer and expression of human blood coagulation factor IX after intramusclular injection of recombinant adeno-associated virus, Proc. Natl. Acad. Sci. USA, 94, 5804, 10.1073/pnas.94.11.5804 Elder, 1996, Successful culture and selection of cytokine gene-modified human dermal fibroblasts for the biologic therapy of patients with cancer, Hum. Gene Ther., 7, 479, 10.1089/hum.1996.7.4-479 Tahara, 1995, IL-12 gene therapy using direct injection of tumors with genetically engineered autologous fibroblasts, Hum. Gene Ther., 6, 1607, 10.1089/hum.1995.6.12-1607 Crystal, 1995, Transfer of genes to humans: early lessons and obstacles to success, Science, 270, 404, 10.1126/science.270.5235.404 Ishibashi, 1993, Hypercholesterolemia in low density lipoprotein receptor knockout mice and its reversal by adenovirus-mediated gene delivery, J. Clin. Invest., 92, 883, 10.1172/JCI116663 Kozarksy, 1994, In vivo correction of low density lipoprotein receptor deficiency in the Watanabe heritable hyperlipidemic rabbit with recombinant adenoviruses, J. Biol. Chem., 269, 13695, 10.1016/S0021-9258(17)36886-2 Bennett, 1996, Photoreceptor cell rescue in retinal degeneration (rd) mice by in vivo gene therapy, Nat. Med., 2, 649, 10.1038/nm0696-649 Zabner, 1993, Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis, Cell, 75, 207, 10.1016/0092-8674(93)80063-K Crystal, 1994, Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis, Nat. Genet., 8, 42, 10.1038/ng0994-42 Knowles, 1995, A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patient with cystic fibrosis, N. Engl. J. Med., 333, 823, 10.1056/NEJM199509283331302 Wolff, 1990, Direct gene transfer into mouse muscle in vivo, Science, 247, 1465, 10.1126/science.1690918 Jiao, 1992, Direct gene transfer into nonhuman primate myofibres in vivo, Hum. Gene Ther., 3, 21, 10.1089/hum.1992.3.1-21 Davis, 1993, Plasmid DNA is superior to viral vectors for direct gene transfer into adult mouse skeletal muscle, Hum. Gene Ther., 4, 733, 10.1089/hum.1993.4.6-733 Wu, 1989, Targeting genes: delivery and persistent expression of a foreign gene driven by mammalian regulatory elements in vivo, J. Biol. Chem., 264, 16985, 10.1016/S0021-9258(18)71447-6 Tsukamoto, 1995, Gene transfer and expression in progeny after intravenous DNA injection into pregnant mice, Nat. Genet., 9, 243, 10.1038/ng0395-243 Caplan, 1995, Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis, Nat. Med., 1, 39, 10.1038/nm0195-39 Cao, 1995, Gene therapy of Parkinson disease model rat by direct injection of plasmid DNA–lipofectin complex, Hum. Gene Ther., 6, 1497, 10.1089/hum.1995.6.11-1497 Nabel, 1994, Safety and toxicity of catheter gene delivery to the pulmonary vasculature in a patient with metastatic melanoma, Hum. Gene Ther., 5, 1089, 10.1089/hum.1994.5.9-1089 Kaneda, 1989, Increased expression of DNA cointroduced with nuclear protein in adult rat liver, Science, 243, 375, 10.1126/science.2911748 Curiel, 1991, Adenovirus enhancement of transferrin-polylysine mediated gene delivery, Proc. Natl. Acad. Sci. USA, 88, 8850, 10.1073/pnas.88.19.8850 Cotten, 1992, High-efficiency receptor-mediated delivery of small and large (48 kb) gene constructs using the endosome-disruption activity of defective or chemically-inactivated adenovirus particles, Proc. Natl. Acad. Sci. USA, 89, 6094, 10.1073/pnas.89.13.6094 Cristiano, 1993, Hepatic gene therapy: adenovirus enhancement of receptor-mediated gene delivery and expression in primary hepatocytes, Proc. Natl. Acad. Sci. USA, 90, 2122, 10.1073/pnas.90.6.2122 Wagner, 1992, Coupling of adenovirus to transferrin-polylysine/DNA complexes greatly enhances receptor-mediated gene delivery and expression of transfected genes, Proc. Natl. Acad. Sci. USA, 89, 6099, 10.1073/pnas.89.13.6099 Wilson, 1992, Hepatocyte-directed gene transfer in vivo leads to transient improvement of hypercholesterolemia in low density lipoprotein-deficient rabbits, J. Biol. Chem., 267, 963, 10.1016/S0021-9258(18)48379-2 Poncet, 1996, Antifection: an antibody-mediated method to introduce genes into lymphoid cells in vitro and in vivo, Gene Ther., 3, 731 Sun, 1998, Microencapsulation of pancreatic islet cells: a bioartificial endocrine pancreas, Methods Enzymol., 137, 575, 10.1016/0076-6879(88)37053-9 Awrey, 1996, Permeability of alginate microcapsules to secretory recombinant gene products, Biotechnol. Bioeng., 52, 472, 10.1002/(SICI)1097-0290(19961120)52:4<472::AID-BIT3>3.0.CO;2-M King, 1987, Alginate–polylysine microcapsules of controlled membrane molecular weight cutoff for mammalian cell culture engineering, Biotechnol. Prog., 3, 231, 10.1002/btpr.5420030407 O'Shea, 1984, Prolonged survival of transplanted islets of Langerhans encapsulated in a biocompatible membrane, Biochim. Biophys. Acta, 804, 133, 10.1016/0167-4889(84)90107-1 Wong, 1991, A novel two step procedure for immobilizing living cells in microcapsules for improving xenograft survival, Biomater. Artif. Cells Immobilization Biotechnol., 19, 687, 10.3109/10731199109117847 Tse, 1996, Secretion of recombinant proteins from hydroxyethyl methacrylate–methyl methacrylate capsules, Biotechnol. Bioeng., 51, 271, 10.1002/(SICI)1097-0290(19960805)51:3<271::AID-BIT3>3.0.CO;2-A Chang, 1993, Delivery of recombinant gene products with microencapsulated cells in vivo, Hum. Gene Ther., 4, 433, 10.1089/hum.1993.4.4-433 C.J.D. Ross, P.L. Chang, Microencapsulation – A novel gene therapy for lysosomal storage diseases, in: W.M. Kuhtreiber, R.P. Lonza, W.L. Chick (Eds.), Handbook of Encapsulated Cell Technology and Therapeutics, Birkhauser, Boston, 1997, chapt. 28. Sassoon, 1993, Myogenic regulatory factors: dissecting their role and regulation during vertebrate embryogenesis, Dev. Biol., 156, 11, 10.1006/dbio.1993.1055 Olson, 1992, Interplay between proliferation and differentiation within the myogenic lineage, Dev. Biol., 154, 261, 10.1016/0012-1606(92)90066-P Crescenzi, 1994, Transformation by myc prevents fusion but not biochemical differentiation of C2C12 myoblasts: mechanisms of phenotypic correction in mixed culture with normal cells, J. Cell Biol., 125, 1137, 10.1083/jcb.125.5.1137 Nguyen, 1983, Reversibility of muscle differentiation in the absence of commitment: analysis of a myogenic cell line temperature-sensitive for commitment, Cell, 34, 281, 10.1016/0092-8674(83)90159-9 K.M. Bowie, P.L. Chang, Differentiation of recombinant myoblasts in alginate microcapsules (in preparation). Devlin, 1983, Re-entry into the cell cycle of differentiated skeletal myocytes, Dev. Biol., 95, 175, 10.1016/0012-1606(83)90016-7 Chamberlain, 1985, Regulation of creatine kinase induction in differentiating mouse myoblasts, Mol. Cell. Biol., 5, 484, 10.1128/MCB.5.3.484 Shainberg, 1971, Alterations of enzymatic activities during muscle differentiation in vitro, Dev. Biol., 25, 1, 10.1016/0012-1606(71)90017-0 Miller, 1992, Myoblast diversity in skeletal myogenesis: how much and to what end?, Cell, 69, 1, 10.1016/0092-8674(92)90111-O Ludolph, 1995, Transcription factor families: muscling in on the myogenic program, FASEB J., 9, 1595, 10.1096/fasebj.9.15.8529839 Blau, 1993, Myoblasts in pattern formation and gene therapy, Trends Genet., 9, 269, 10.1016/0168-9525(93)90012-7 Mangan, 1996, A muscle-specific variant of microtubule-associated protein 4 (MAP4) is required in myogenesis, Development, 122, 771, 10.1242/dev.122.3.771 Seigneurin-Venin, 1996, Involvement of the dihydropyridine receptor and internal Ca2+ stores in myoblast fusion, Exp. Cell Res., 223, 301, 10.1006/excr.1996.0085 Peck, 1993, Differential effects of over-expressed neural cell adhesion molecule isoforms on myoblast fusion, J. Cell Biol., 123, 1587, 10.1083/jcb.123.6.1587 Zeschnigk, 1995, Involvement of M-cadherin in terminal differentiation of skeletal muscle cells, J. Cell Sci., 108, 2973, 10.1242/jcs.108.9.2973 Fazeli, 1996, Altered secondary myogenesis in transgenic animals expressing the neural cell adhesion molecule under the control of a skeletal muscle-actin promoter, J. Cell Biol., 135, 241, 10.1083/jcb.135.1.241 Hortelano, 1996, Delivery of human factor IX in mice by encapsulated recombinant myoblasts: a novel approach towards allogeneic gene therapy of Hemophilia B, Blood, 87, 5095, 10.1182/blood.V87.12.5095.bloodjournal87125095 Lin, 1992, Pit-1-dependent expression of the receptor for growth hormone releasing factor mediates pituitary cell growth, Nature, 360, 767, 10.1038/360765a0 Al-Hendy, 1995, Allogeneic somatic gene therapy: correction of growth hormone deficiency in mice with microencapsulated non-autologous myoblasts, Hum. Gene Ther., 6, 165, 10.1089/hum.1995.6.2-165