Delivery of recombinant gene products to the central nervous system with nonautologous cells in alginate microcapsules

Human Gene Therapy

Volumn 10, Issue 1, 1999, Pages 49-59

  Ross, C J D ^a   Ralph, M ^b   Chang, P L ^a  

^a MCMASTER UNIVERSITY   (Canada)

^b UNIVERSITY OF TORONTO   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

ALGINIC ACID; HUMAN GROWTH HORMONE; POLYLYSINE;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; BRAIN; BRAIN VENTRICLE; CENTRAL NERVOUS SYSTEM; CONTROLLED STUDY; GENE TARGETING; GROWTH HORMONE RELEASE; IMMUNOHISTOCHEMISTRY; MARKER GENE; MICROCAPSULE; MOUSE; MYOBLAST; NONHUMAN; RECOMBINANT GENE;

ALGINATES; ANIMALS; BIOCOMPATIBLE MATERIALS; BIOLOGICAL MARKERS; BLOOD-BRAIN BARRIER; BRAIN; CAPSULES; CELL LINE; DNA, RECOMBINANT; GENE EXPRESSION; GENE TRANSFER TECHNIQUES; GENES, REPORTER; HUMAN GROWTH HORMONE; HUMANS; IMMUNOHISTOCHEMISTRY; MICE; MODELS, BIOLOGICAL; TIME FACTORS;

EID: 0032928402     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/10430349950019183     Document Type: Article

References (42)

1. AEBISCHER, P., TRESCO, P.A., WINN, S.R., GREEN, L.A., and JAEGER, C.B. (1991). Long-term cross-species brain transplantation of a polymer-encapsulated dopamine-secreting cell line. Exp. Neurol. 111, 269-275.
2. 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. Nature Med. 2, 696-699.
3. 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, 165-175.
4. BASTEDO, L., ROSS, C.J.D., HORTELANO, G., and CHANG, P.L. (1999). Allogeneic gene therapy on the murine model of mucopolysaccharidosis type VII. (Submitted.)
5. CHANG, P.L. (1997). Microcapsules as bio-organs for somatic gene therapy. Ann. N.Y. Acad. Sci. 831, 461-473.
6. CHANG, P.L., and BOWIE, K. (1998). Development of engineered cells for implantation in gene therapy. Adv. Drug Deliv. Rev. (in press)
7. CHANG, P.L., CAPONE, J.P., and BROWN, G.M. (1990). Autologous fibroblast implantation. Feasibility and potential problems in gene replacement therapy. Mol. Biol. Med. 7, 461-470.
8. CHANG, P.L., SHEN, N., and WESTCOTT, A.J. (1993). Delivery of recombinant gene products with microencapsulated cells in vivo. Hum. Gene Ther. 4, 433-440.
9. CHANG, T.M.S. (1964). Semipermeable capsules. Science 146, 524-525.
10. CUNNINGHAM, L.A., SHORT, M.P., VIELKIND, U., BREAKEFIELD, X.O., and BOHN, M.C. (1991). Survival and differentiation within the adult mouse striatum of grafted rat pheochromocytoma cells (PC 12) genetically modified to express recombinant beta-NGF. Exp. Neurol. 112, 174-182.
11. DOERING, L.C., and CHANG, P.L. (1991). Expression of a novel gene product by transplants of genetically modified primary fibroblasts in the central nervous system. J. Neurosci. Res. 29, 292-298.
12. EMERICH, D.F., McDERMOTT, P.E., KRUEGER, P.M., FRYDEL, B., SANBERG, P.R., and WINN, S.R. (1993). Polymer-encapsulated PC 12 cells promote recovery of motor function in aged rats. Exp. Neurol. 122, 37-47.
13. EMERICH, D.F., HAMMANG, J.P., BAETGE, E.E., and WINN, S.R. (1994a). Implantation of polymer-encapsulated human nerve growth factor-secreting fibroblasts attenuates the behavioral and neuropathological consequences of quinolinic acid injections into rodent striatum. Exp. Neurol. 130, 141-150.
14. EMERICH, D.F., WINN, S.R., HARPER, J., HAMMANG, J.P., BAETGE, E.E., and KORDOWER, J.H. (1994b). Implants of polymer-encapsulated human NGF-secreting cells in the nonhuman primate: Rescue and sprouting of degenerating cholinergic basal forebrain neurons. J. Comp. Neurol. 349, 148-164.
15. GAGE, F.H., WOLFF, J.A., ROSENBERG, M.B., XU, L., YEE, J.K., SHULTS, C., and FRIEDMANN, T. (1987). Grafting genetically modified cells to the brain: Possibilities for the future. Neuroscience 23, 795-807.
16. GOLDSTEIN, A., ARONOW, L., and KALMAN, S.M. (1974). Principles of Drug Action, 2nd Ed. (John Wiley & Sons, New York) pp. 301-355.
17. HATTON, J.D., LECHTMAN, A.N., and U, H.S. (1992). Formation of PC12 tumors after transplantation into rat brains: Dependence of time course on host age. Cancer Res. 52, 1933-1937.
18. HOFFMAN, D., BREAKEFIELD, X.O., SHORT, M.P., and AEBISCHER, P. (1993). Transplantation of a polymer-encapsulated cell line genetically engineered to release NGF. Exp. Neurol. 122, 100-106.
19. HORTELANO, G., AL-HENDY, A., OFOSU, F.A., and CHANG, P.L. (1996). Delivery of human factor IX in mice by encapsulated recombinant myoblasts: A novel approach towards allogeneic gene therapy of hemophilia B. Blood 87, 5095-5103.
20. HUGES, M., VASSILAKOS, A., ANDREW, 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, 1445-1455.
21. JAEGER, C.B. (1985). Immunocytochemical study of PC12 cells grafted to the brain of immature rats. Exp. Brain Res. 59, 615-624.
22. KORDOWER, J.H., WINN, S.R., LIU, Y.T., MUFSON, E.J., SLADEK, J.R., JR., HAMMANG, J.P., BAETGE, E.E., and EMERICH, D.F. (1994). The aged monkey basal forebrain: Rescue and sprouting of axotomized basal forebrain neurons after grafts of encapsulated cells secreting human nerve growth factor. Proc. Natl. Acad. Sci. U.S.A. 91, 10898-10902.
23. LEDLEY, T.S., and LEDLEY, F.D. (1994). Multicompartment, numerical model of cellular events in the pharmacokinetics of gene therapies. Hum. Gene Ther. 5, 679-691.
24. LIM, F., and SUN, A.M. (1980). Microencapsulated islets as bioartificial endocrine pancreas. Science 210, 908-910.
25. LIU, H.W., OFOSU, F.A., and CHANG, P.L. (1993). Expression of human factor IX by microencapsulated recombinant fibroblasts. Hum. Gene Ther. 4, 291-301.
26. LOWRY, O.H., ROSENBROUGH, N.J., FAR, A.L., and RANDAL, R.J. (1951). Protein measurement with the folin phenol reagent. J. Biol. Chem. 293, 265-275.
27. MIYAZONO, M., LEE, V.M., and TROJANOWSKI, J.Q. (1995). Proliferation, cell death, and neuronal differentiation in transplanted human embryonal carcinoma (NTera2) cells depend on the graft site in nude and severe combined immunodeficient mice. Lab. Invest. 73, 273-283.
28. O'SHEA, G.M., and SUN, A.M. (1986). Encapsulation of rat islets of Langerhans prolongs xenograft survival in diabetic mice. Diabetes 35, 943-946.
29. O'SHEA, G.M., GOOSEN, M.F., and SUN, A.M. (1984). Prolonged survival of transplanted islets of Langerhans encapsulated in a biocompatible membrane. Biochim. Biophys. Acta 804, 133-136.
30. SAGEN, J., WANG, H., TRESCO, P.A., and AEBISCHER, P. (1993). Transplants of immunologically isolated xenogeneic chromaffin cells provide a long-term source of pain-reducing neuroactive substances. J. Neurosci. 13, 2415-2423.
31. SAGOT, Y., TAN, S.A., BAETGE, E., SCHMALBRUCH, H., KATO, A.C., and AEBISCHER, P. (1995). Polymer encapsulated cell lines genetically engineered to release ciliary neurotrophic factor can slow down progressive motor neuronopathy in the mouse. Eur. J. Neurosci. 7, 1313-1322.
32. SAGOT, Y., TAN, S.A., HAMMANG, J.P., AEBISCHER, P., and KATO, A.C. (1996). GDNF slows loss of motoneurons but not axonal degeneration or premature death of pmn/pmn mice. J. Neurosci. 16, 2335-2341.
33. SAITOH, Y., TAKI, T., ARITA, N., OHNISHI, T., and HAYAKAWA, T. (1995). Cell therapy with encapsulated xenogeneic tumor cells secreting beta-endorphin for treatment of peripheral pain. Cell Transplant. 4(Suppl. 1), S13-7.
34. SCHINSTINE, M., FIORE, D.M., WINN, S.R., and EMERICH, D.F. (1995). Polymer-encapsulated Schwannoma cells expressing human nerve growth factor promote the survival of cholinergic neurons after a fimbria-fornix transection. Cell Transplant. 4, 93-102.
35. STOCKLEY, T.L., and CHANG, P.L. (1997). Non-autologous somatic gene therapy: A novel approach to gene therapy. Drug News Perspect. 10, 331-340.
36. SUN, A.M. (1988). Microencapsulation of pancreatic islet cells: A bioartificial endocrine pancreas. Methods Enzymol. 137, 575-580.
37. SUN, Y., MA, X., ZHOU, D., VACEK, I., and SUN, A.M. (1996). Normalization of diabetes in spontaneously diabetic cynomologus monkeys by xenografts of microencapsulated porcine islets without immunosuppression. J. Clin. Invest. 98, 1417-1422.
38. TRESCO, P.A., WINN, S.R., TAN, S., JAEGER, C.B., GREENE, L.A., and AEBISCHER, P. (1992). Polymer-encapsulated PC12 cells: Long-term survival and associated reduction in lesion-induced rotational behavior. Cell Transplant. 1, 255-264.
39. TURYN, D., and BARTKE, A. (1993). Pharmacokinetics of radioiodinated human and ovine growth hormones in transgenic mice expressing bovine growth hormone. Transgenic Res. 2, 219-226.
40. WINN, S.R., TRESCO, P.A., ZIELINSKI, B., GREENE, L.A., JAEGER, C.B., and AEBISCHER, P. (1991). Behavioral recovery following intrastriatal implantation of microencapsulated PC12 cells. Exp. Neurol. 113, 322-329.
41. WINN, S.R., HAMMANG, I.P., EMERICH, D.F., LEE, A., PALMITER, R.D., and BAETGE, E.E. (1994). Polymer-encapsulated cells genetically modified to secrete human nerve growth factor promote the survival of axotomized septal cholinergic neurons. Proc. Natl. Acad. Sci. U.S.A. 91, 2324-2328.
42. WINN, S.R., LINDNER, M.D., LEE, A., HAGGETT, G., FRANCIS, J.M., and EMERICH, D.F. (1996). Polymer-encapsulated genetically modified cells continue to secrete human nerve growth factor for over one year in rat ventricles: Behavioral and anatomical consequences. Exp. Neurol. 140, 126-138.