Long-term in vitro correction of α-L-iduronidase deficiency (Hurler syndrome) in human bone marrow

Proceedings of the National Academy of Sciences of the United States of America

Volumn 93, Issue 5, 1996, Pages 2025-2030

  Fairbairn, L J ^a   Lashford, L S ^a   Spooncer, E ^a   Mcdermott, R H ^a   Lebens, G ^a   Arrand, J E ^a   Arrand, J R ^a   Bellantuono, I ^a   Holt, R ^a   Hatton, C E ^b   Cooper, A ^b   Besley, G T N ^b   Wraith, J E ^b   Anson, D S ^c   Hopwood, J J ^c   Dexter, T M ^a  

^a CHRISTIE HOSPITAL   (United Kingdom)

^b ROYAL MANCHESTER CHILDREN S HOSPITAL   (United Kingdom)

^c WOMEN S AND CHILDREN S HOSPITAL   (Australia)

Author keywords

autosomal recessive; gene therapy; mucopolysaccharidosis type 1

Indexed keywords

CD34 ANTIGEN; COMPLEMENTARY DNA; DERMATAN SULFATE; GLYCOSAMINOGLYCAN; HEPARAN SULFATE; LEVO IDURONIDASE; VIRUS VECTOR;

ARTICLE; AUTOSOMAL RECESSIVE DISORDER; BONE MARROW CELL; CLINICAL PROTOCOL; CONTROLLED STUDY; ENZYME DEFICIENCY; GENE THERAPY; GENE TRANSFER; HEMATOPOIETIC CELL; HUMAN; HUMAN CELL; HURLER SYNDROME; LYSOSOME STORAGE DISEASE; MUCOPOLYSACCHARIDOSIS; PRIORITY JOURNAL;

ANTIGENS, CD34; BASE SEQUENCE; BONE MARROW; CELLS, CULTURED; DNA PRIMERS; GENE EXPRESSION; GENE THERAPY; GENETIC VECTORS; HEMATOPOIETIC STEM CELLS; HUMANS; IDURONIDASE; MOLECULAR SEQUENCE DATA; MUCOPOLYSACCHARIDOSIS I; PHENOTYPE; TIME FACTORS;

EID: 9044223652     PISSN: 00278424     EISSN: None     Source Type: Journal    
DOI: 10.1073/pnas.93.5.2025     Document Type: Article

References (35)

1. Hopwood, J. J. & Morris, C. P. (1990) Mol. Biol. Med. 7, 381-404.
2. Neufeld, E. F. & Meunzer, J. (1989) in The Metabolic Basis of Inherited Disease, eds. Beandet, A. L., Sly, W. S. & Valle, D. (McGraw Hill, New York), pp. 1565-1587.
3. Whitley, C.B. (1993) in Inheritable Disorders of Connective Tissue, ed. Beighton, P. (Mosby, St. Louis), pp. 367-499.
4. Ashton, L. J., Brooks, D. A., McCourt, P.A., Muller, V. J., Clements, P. R. & Hopwood, J. J. (1992) Am. J. Hum. Genet. 50, 787-794.
5. Hopwood, J. J., Vellodi, A., Scott, H. S., Morris, C. P., Litjens, T., Clements, P. R., Brooks, D. A., Cooper, A. & Wraith, J. E. (1993) J. Inherit. Metab. Dis. 16, 1024-1033.
6. Whitley, C. B., Belani, K. G., Chang, P. N., Summers, C. G., Blazar, B. R., Tsai, M. Y., Latchaw, R. E., Ramsay, N. K. & Kersey, J. H. (1993) Am. J. Med. Genet. 46, 209-218.
7. Hobbs, J. R., Hugh-Jones, K., Barret, A. J., Byrom, N., Chambers, D., Henry, K., James, D. C. O., Lucas, C. F., Benson, P. F., Tansley, L. R., Patrick, A. D., Mossman, J. & Young, E. P. (1981) Lancet, ii, 709-712.
8. Whitley, C. B., Ramsey, N. K. C., Kersey, J. H., Belani, K. G., Tsai, M. Y., Sibley, R. K., Snover, D. C. & Krivit, W. (1986) in Bone Marrow Transplantation for Treatment of Lysosomal Storage Diseases, eds. Krivit, W. & Paul, N. W. (Liss, New York), pp. 7-24.
9. Scott, H. S., Anson, D. S., Osborne, A. M., Nelson, P. V., Clements, P. R., Morris, C. P. & Hopwood, J. J. (1991) Proc. Natl. Acad. Sci. USA 88, 9695-9699.
10. Scott, H. S., Guo, X. H., Hopwood, J. J. & Morris, C. P. (1992) Genomics 13, 1311-1313.
11. Anson, D. S., Bielicki, J. & Hopwood, J. J. (1992) Hum. Gene Ther. 3, 371-379.
12. Shull, R., Kakkis, E., McEntee, M., Kania, S., Jonas, A. & Neufeld, E. (1994) Proc. Natl. Acad. Sci. USA 91, 12937-12941.
13. Miller, A. D. & Rossman, G. J. (1989) BioTechniques 7, 980-990.
14. Markowitz, D., Goff, S. & Bank, A. (1988) Virology 167, 400-406.
15. Fairbairn, L. J. & Spooncer, E. (1993) in Haemopoiesis: A Practical Approach, eds. Testa, N. G. & Molineux, G. (Oxford Univ. Press, Oxford), pp. 175-188.
16. Coutinho, L. H., Gilleece, M. H., DeWynter, E. A. & Testa, N. G. (1993) in Haemopoiesis: A Practical Approach, eds. Testa, N. G. & Molineux, G. (Oxford Univ. Press, Oxford), pp. 75-106.
17. Southern, E. M. (1975) J. Mol. Biol. 98, 503-517.
18. Church, G. M. & Gilbert, W. (1984) Proc. Natl. Acad. Sci. USA 81, 1991-1995.
19. Stirling, J. L., Robinson, D., Fensom, A. H., Benson, P. F. & Baker, J. E. (1978) Lancet i, 147.
20. Krall, W. J., Challita, P. M., Perlmutter, L. S., Skelton, D. C. & Kohn, D. B. (1994) Blood 83, 2737-2748.
21. Wolfe, J. H., Schuchman, E. H., Stramm, L. E., Concaugh, E. A., Haskins, M. E., Aguirre, G. D., Patterson, D. F., Desnick, R. J. & Gilboa, E. (1990) Proc. Natl. Acad. Sci. USA 87, 2877-2881.
22. Braun, S. E., Aronovich, E. L., Anderson, R. A., Crotty, P. L., McIvor, R. S. & Whitley, C. B. (1993) Proc. Natl. Acad. Sci. USA 90, 11830-11834.
23. Anson, D. S., Muller, V., Bielicki, J., Harper, G. S. & Hopwood, J. J. (1993) Biochem. J. 294, 657-662.
24. Moullier, P., Bohl, D., Heard, J. M. & Danos, O. (1993) Nat. Genet. 4, 154-159.
25. Marechal, V., Naffakh, N., Danos, O. & Heard, J. M. (1993) Blood 82, 1358-1365.
26. Wolfe, J. H., Sands, M. S., Barker, J. E., Gwynn, B., Rowe, L. B., Vogler, C. A. & Birkenmeier, E. H. (1992) Nature (London) 360, 749-753.
27. Miller, D. G., Adam, M. A. & Miller, A. D. (1990) Mol. Cell. Biol. 10, 4239-4242.
28. Lord, B. I. & Woolford, L. B. (1993) Stem Cells Dayton 11, 212-217.
29. Sutherland, H. J., Lansdorp, P.M., Henkelman, D. H., Eaves, A. C. & Eaves, C. J. (1990) Proc. Natl. Acad. Sci. USA 87, 3584-3588.
30. Sutherland, H. J., Eaves, C. J., Lansdorp, P. M., Thacker, J. D. & Hogge, D. E. (1991) Blood 78, 666-672.
31. Chang, J., Morgenstern, G. R., Coutinho, L. H., Scarffe, J. H., Carr, T., Deakin, D. P., Testa, N. G. & Dexler, T. M. (1989) Bone Marrow Transpl. 4, 5-9.
32. Chang, J., Coutinho, L., Morgenstern, G., Scarffe, J. H., Deakin, D., Harrison, C., Testa, N. G. & Dexter, T. M. (1986) Lancet i, 294-295.
33. Moore, K. A., Deisseroth, A. B., Reading, C. L., Williams, D. E. & Belmont, J. W. (1992) Blood 79, 1393-1399.
34. Hughes, P. F., Thacker, J. D., Hogge, D., Sutherland, H. J., Thomas, T. E., Lansdorp, P. M., Eaves, C. J. & Humphries, R. K. (1992) J. Clin. Invest. 89, 1817-1824.
35. Nolta, J. A., Yu, X. J., Bahner, I. & Kohn, D. B. (1992) J. Clin. Invest. 90, 342-348.