Preclinical studies of lymphocyte gene therapy for mild Hunter syndrome (mucopolysaccharidosis type II)

Human Gene Therapy

Volumn 7, Issue 3, 1996, Pages 283-290

  Braun, Stephen E ^a,b,c   Pan, Dao ^a,b   Aronovich, Elena L ^a,b   Jonsson, Jon J ^a,b,d   McIvor, R Scott ^a,b   Whitley, Chester B ^a,b,e  

^a UNIVERSITY OF MINNESOTA   (United States)

^b UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

^c INDIANA UNIVERSITY SCHOOL OF MEDICINE   (United States)

^d YALE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^e UNIV OF MINNESOTA HOSP AND CLINIC   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA; GLYCOSAMINOGLYCAN; IDURONATE 2 SULFATASE; INTERLEUKIN 2; MONOCLONAL ANTIBODY CD3;

ARTICLE; CELL CULTURE; CONTROLLED STUDY; CULTURE MEDIUM; CYTOMEGALOVIRUS; ENZYME ACTIVITY; GENE THERAPY; GENE TRANSFER; HUMAN; HUMAN CELL; HUNTER SYNDROME; LONG TERMINAL REPEAT; LYMPHOCYTE; LYMPHOCYTE ACTIVATION; MOLONEY LEUKEMIA ONCOVIRUS; PROMOTER REGION; RETROVIRUS; T LYMPHOCYTE; TRANSCRIPTION REGULATION;

CYTOMEGALOVIRUS; MOLONEY MURINE LEUKEMIA VIRUS; MURINAE; ONCOVIRINAE; UNIDENTIFIED RETROVIRUS;

EID: 0030060184     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.1996.7.3-283     Document Type: Article

References (42)

1. ADINOLFI, M., and BROWN, S. (1984). Strategies for the correction of enzymatic deficiencies in patients with mucopolysaccharidoses. Dev. Med. Child Neural. 26, 404-408.
2. BIRKENMEIER, E.H., BARKER, J.E., VOGLER, C.A., KYLE, J.W., SLY, W.S., GWYNN, B., LEVY, B., and PEGORS, C. (1991). Increased life span and correction of metabolic defects in murine mucopolysaccharidosis type VII after syngeneic bone marrow transplantation. Blood 78, 3081-3092.
3. BLAESE, R.M. (1993). Development of gene therapy for immunodeficiency: adenosine deaminase deficiency. Pediatr. Res. 33, S49-S55.
4. BRADFORD, M.M. (1976). A rapid and sensitive method for the quantitation of microgram quantities of protein utilizing the principle of protein-dye binding. Anal. Biochem. 72, 248-254.
5. BRAUN, S., ARONOVICH, E., ANDERSON, R., CROTTY, P., McIVOR, R.S., and WHITLEY, C. (1993). Metabolic correction and cross-correction of mucopolysaccharidosis type II (Hunter syndrome) by retroviral-mediated gene transfer and expression of human iduronate-2-sulfatase. Proc. Natl. Acad. Sci. USA 90, 11830-11834.
6. CULVER, K., CORNETTA, K., MORGAN, R., MORECKI, S., AEBERSOLD, P., KASID, A., LOTZE, M., ROSENBERG, S.A., ANDERSON, W.F., and BLAESE, R.M. (1991). Lymphocytes as cellular vehicles for gene therapy in mouse and man. Proc. Natl. Acad. Sci. USA 88, 3155-3159.
7. DI FERRANTE, N., NICHOLS, B.I., DONNELLY, P.V., NERI, G., HRGOVCIC, R., and BERGLUND, R.K. (1971). Induced degradation of glycosaminoglycans in Hurler's and Hunter's syndromes by plasma infusion. Proc. Natl. Acad. Sci. USA 68, 303-307.
8. FENJVES, E.S., BLAESE, R.M., and TAICHMAN, L.B. (1995). Cutaneous gene therapy for ADA deficiency: A model approach for inherited metabolic disorders. J. Cell. Biochem. 21A, 363.
9. FRATANTONI, J.C., HALL, C.W., and NEUFELD, E.F. (1968). Hurler and Hunter syndromes: Mutual correction of the defect in cultured fibroblasts. Science 162, 699-706.
10. GASPER, P.W., THRALL, M.A., WENGER, D.A., MACY, D.W., HAM, L., DORNSIFE, R.E., MCBILES, K., QUACKENBUSH, S.L., KESEL, M.L., GILLETTE, E.L., et al. (1984). Correction of feline arylsulphatase B deficiency (mucopolysaccharidosis VI) by bone marrow transplantation. Nature 312, 467-469.
11. HICKMAN, S., and NEUFELD, E.F. (1972). A hypothesis for I-cell disease: defective hydrolases that do not enter lysosomes. Biochem. Biophys. Res. Commun. 49, 992-999.
12. HOBBS, J.R. (1981). Bone marrow transplantation for inborn errors. Lancet 2, 735-739.
13. HOBBS, J.R., HUGH-JONES, K., BARRETT, A.J., BYROM, N., CHAMBERS, D., HENRY, K., JAMES, D.C., LUCAS, C.F., ROGERS, T.R., BENSON, P.F., TANSLEY, L.R., PATRICK, A.D., MOSSMAN, J., and YOUNG, E.P. (1981). Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation. Lancet 2, 709-712.
14. HOPWOOD, J.J., and MORRIS, C.P. (1990). The mucopolysaccharidoses. Diagnosis, molecular genetics and treatment. Mol. Biol. Med. 7, 381-404.
15. JONSSON, J.J., BRAUN, S.E., PAN, D., ARONOVICH, E.L., McIVOR, R.S., and WHITLEY, C.B. (1993). In vitro correction of iduronate-2-sulfatase deficiency in Hunter syndrome (mucopolysaccharidosis type II) with four retroviral vectors (abstract). Am. J. Hum. Genet. 53, 409.
16. JONSSON, J.J., ARONOVICH, E.L., BRAUN, S.E., and WHITLEY, C.B. (1995). Molecular diagnosis of mucopolysaccharidosis type II (Hunter syndrome) by automated sequencing and computer-assisted interpretation: toward mutation mapping of the iduronate-2-sulfatase gene. Am. J. Hum. Genet. 56, 597-607.
17. KNUDSON, A.G., DI FERRANTE, N., and CURTIS, J.E. (1971). The effect of leukocyte transfusion in a child with type II mucopolysaccharidosis. Proc. Natl. Acad. Sci. USA 1738-1741.
18. + cells purified from peripheral blood of breast cancer patients primed with chemotherapy and granulocyte-macrophage colony-stimulating factor. Hum. Gene Ther. 5, 203-208.
19. MILLER, A.D., and ROSMAN, G.J. (1989). Improved retroviral vectors for gene transfer and expression. Biotechniques 7, 980-982.
20. MUENZER, J., NEUFELD, E.F., CONSTANTOPOULOS, G., CARUSO, R.C., KAISER, K.M., PIKUS, A., DANOFF, J., BERRY, R.R., MCDONALD, H.D., and THOMPSON, J.N. (1992). Attempted enzyme replacement using human amnion membrane implantations in mucopolysaccharidoses. J. Inherit. Metab. Dis. 15, 25-37.
21. MULLEN, C.A., SNITZER, K., and BLAESE, R.M. (1994). Long term in vivo expression of lymphocyte clones from an ADA-SCID patient treated with gene therapy. J. Cell. Biochem. 18A, 240.
22. NEUFELD, E.F. (1991). Lysosomal storage diseases. Annu. Rev. Biochem. 60, 257-280.
23. NEUFELD, E.F., and MUENZER, J. (1989). The mucopolysaccharidoses. In The Metabolic Basis of Inherited Diseases, 6th ed. C.R. Scriver, A.L. Beaudet, W.S. Sly, and D. Valle, eds. (McGraw-Hill, New York) pp. 1565-1587.
24. OLSEN, I., DEAN, M.F., HARRIS, G., and MUIR, H. (1981). Direct transfer of a lysosomal enzyme from lymphoid cells to deficient fibroblasts. Nature 291, 244-247.
25. PAN, D., SHANKAR, R., STRONCEK, D.F., McCULLOUGH, J., McIVOR, R.S., and WHITLEY, C.B. (1995). Retrovirus production and transduction of peripheral blood lymphocytes in a closed artificial capillary system: an approach to large scale cell manipulation for clinical gene therapy (abstract). J. Cell. Biochem. 21A, 412.
26. PARKMAN, R., BARRANTES, B., WEINBERG, K.I., HEISS, L., and KOHN, D. (1995). Analysis of T lymphocytes from patients with adenosine deaminase (ADA) deficiency transplanted with ADA transduced cord blood cells. J. Cell. Biochem. 21A, 369.
27. RESNICK, J.M., KRIVIT, W., SNOVER, D.C., KERSEY, J.H., RAMSAY, N.K., BLAZAR, B.R., and WHITLEY, C.B. (1992). Pathology of the liver in mucopolysaccharidosis: Light and electron microscopic assessment before and after bone marrow transplantation. Bone Marrow Transpl. 10, 273-280.
28. RIVERO, J.L., LACORAZZA, H.D., KOZHICH, A., NUSSENBLATT, R.B., and JENDOUBI, M. (1994). Retrovirus-mediated gene transfer and expression of human ornithine delta-aminotransferase into embryonic fibroblasts. Hum. Gene Ther. 5, 701-707.
29. SANDS, M.S., VOGLER, C., KYLE, J.W., GRUBB, J.H., LEVY, B., GALVIN, N., SLY, W.S., and BIRKENMEIER, E.H. (1994). Enzyme replacement therapy for murine mucopolysaccharidosis type VII. J. Clin. Invest. 93, 2324-2331.
30. SHULL, R.M., and WALKER, M.A. (1988). Radiographic findings in a canine model of mucopolysaccharidosis I. Changes associated with bone marrow transplantation. Invest. Radiol. 23, 124-130.
31. SHULL, R.M., HASTINGS, N.E., SELCER, R.R., JONES, J.B., SMITH, J.R., CULLEN, W.C., and CONSTANTOPOULOS, G. (1987). Bone marrow transplantation in canine mucopolysaccharidosis I. Effects within the central nervous system. J. Clin. Invest. 79, 435-443.
32. SHULL, R.M., BREIDER, M.A., and CONSTANTOPOULOS, G.C. (1988). Long-term neurological effects of bone marrow transplantation in a canine lysosomal storage disease. Pediatr. Res. 24, 347-352.
33. VARKI, A., and KORNFELD, S. (1981). Lysosomal enzyme targeting: N-acetylglucosaminyl-phospho-transferase selectively phosphorylated native lysosomal enzymes. J. Biol. Chem. 256, 11977-11980.
34. WALTERS, L.B. (1995). Department of Health and Human Services National Institutes of Health Recombinant DNA Advisory Committee minutes of meeting September 12-13, 1994. Hum. Gene Ther. 6, 481-525.
35. WARKENTIN, P.I., DIXON, M.S., SCHAFEY, I., STRANDJORD, S.E., and COCCIA, P.F. (1986). Bone marrow transplantation in Hunter syndrome: a preliminary. Birth Defects 22, 31-39.
36. WHITLEY, C.B. (1993). The mucopolysaccharidoses. In McKusick's Heritable Diseases of Connective Tissue, 5th ed. P. Beighton, ed. (Mosby, St. Louis) pp. 367-499.
37. WHITLEY, C.B., RAMSAY, N.K.C., KERSEY, J.H., and KRIVIT, W. (1986). Bone marrow transplantation for Hurler syndrome: assessment of metabolic correction. Birth Defects 22, 7-24.
38. WHITLEY, C.B., BELANI, K.G., CHANG, P.-N., SUMMERS, C.G., BLAZAR, B.R., TSAI, M.Y., LATCHAW, R.E., RAMSAY, N.K.C., and KERSEY, J.H. (1993a). Long-term outcome of Hurler syndrome following bone marrow transplantation. Am. J. Med. Genet. 46, 209-218.
39. WHITLEY, C.B., KRIVIT, W., RAMSAY, N.K.C., KERSEY, J.H., CHANG, P.N., LATCHAW, R.E., BLAZAR, B.R., SCOTT, H.S., NELSON, P.V., MORRIS, C.P., and HOPWOOD, J.J. (1993b). Mutation analysis and clinical outcome of patients with Hurler syndrome (mucopolysaccharidosis type I-H) undergoing bone marrow transplantation. Am. J. Hum. Genet. 53, 101.
40. YOUNG, I.D., and HARPER, P.S. (1982). Mild form of Hunter's syndrome: Clinical delineation based on 31 cases. Arch. Dis. Child. 57, 828-836.
41. YOUNG, I.D., and HARPER, P.S. (1983). The natural history of the severe form of Hunter's syndrome: A study based on 52 cases. Dev. Med. Child Neurol. 25, 481-489.
42. YOUNG, I.D., HARPER, P.S., NEWCOMBE, R.G., and ARCHER, I.M. (1982). A clinical and genetic study of Hunter's syndrome. 2. Differences between the mild and severe forms. J. Med. Genet. 19, 408-411.