Rescue of ATPa3-deficient murine malignant osteopetrosis by hematopoietic stem cell transplantation in utero

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

Volumn 102, Issue 41, 2005, Pages 14629-14634

  Frattini, Annalisa ^a   Blair, Harry C ^b   Sacco, Maria Grazia ^a   Cerisoli, Francesco ^a   Faggioli, Francesca ^a   Catò, Enrica Mira ^a   Pangrazio, Alessandra ^a   Musio, Antonio ^a   Rucci, Francesca ^a   Sobacchi, Cristina ^a   Sharrow, Allison C ^b   Kalla, Sara E ^b   Bruzzone, Maria Grazia ^c   Colombo, Roberte ^d   Magli, Maria Cristina ^a   Vezzoni, Paolo ^a   Villa, Anna ^a,b  

^a CNR   (Italy)

^b UNIVERSITY OF PITTSBURGH SCHOOL OF MEDICINE   (United States)

^c DEPARTMENT OF NEUROSURGERY   (Italy)

^d UNIVERSITÀ CATTOLICA DEL SACRO CUORE   (Italy)

Author keywords

Autosomal recessive osteopetrosis; Bone marrow transplant; Prenatal therapy

Indexed keywords

ADENOSINE TRIPHOSPHATASE;

ALBERS SCHOENBERG DISEASE; ANIMAL CELL; ANIMAL EXPERIMENT; ARTICLE; AUTOSOMAL RECESSIVE DISORDER; BONE MARROW TRANSPLANTATION; BONE REMODELING; CARTILAGE; CELL DIFFERENTIATION; CONTROLLED STUDY; FETUS; GRAFT VERSUS HOST REACTION; HEMATOPOIETIC STEM CELL; HEMATOPOIETIC STEM CELL TRANSPLANTATION; IMMUNOSUPPRESSIVE TREATMENT; MINERALIZATION; MOUSE; NONHUMAN; OSTEOCLAST; PHENOTYPE; PRENATAL CARE; PRIORITY JOURNAL; TRANSGENIC MOUSE;

ANIMALS; BONE MATRIX; ENZYME-LINKED IMMUNOSORBENT ASSAY; FEMALE; FETAL THERAPIES; GREEN FLUORESCENT PROTEINS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; MICE; MICE, TRANSGENIC; OSTEOCLASTS; OSTEOPETROSIS; PREGNANCY; VACUOLAR PROTON-TRANSLOCATING ATPASES;

MURINAE; MUS MUSCULUS;

EID: 26844488044     PISSN: 00278424     EISSN: None     Source Type: Journal    
DOI: 10.1073/pnas.0507637102     Document Type: Article

References (37)

1. Coccia, P. F., Krivit, W., Cervenka, J., Clawson, C., Kersey, J. H., Kim, T. H., Nesbit, M. E., Ramsay, N. K., Warkentin, P. I., Teitelbaum, S. L., et al. (1980) N. Engl. J. Med. 302, 701-708.
2. Pirovano, S., Notarangelo, L. D., Malacarne, F., Mazzolari, E., Porta, F., Lanfranchi, A., Giuliani, S., Zucca, S., Pecorelli, S., Albertini, A., et al. (2004) Haematologica 89, 450-461.
3. Flake, A. W., Roncarolo, M. G., Puck, J. M., Almeida-Porada, G., Evans, M. I., Johnson, M. P., Abella, E. M., Harrison, D. D. & Zanjani, E. D. (1996) N. Engl. J. Med. 335, 1806-1810.
4. Wengler, G. S., Lanfranchi, A., Frusca, T., Verardi, R., Neva, A., Brugnoni, D., Giliani, S., Fiorini, M., Mella, P., Guandalini, F., et al. (1996) Lancet 348, 1484-1487.
5. Gil, J., Porta, F., Bartolome, J., Lanfranchi, A., Verardi, R., Notarangelo, L. D., Carlo-Stella, C., Rodriguez, R., Rodriguez, J. J., Gurbindo, D., et al. (1999) Transplant. Proc. 31, 2581.
6. Bartolome, J., Porta, F., Lafranchi, A., Rodriguez-Molina, J. J., Cela, E., Cantalejo, A., Fernandez-Cruz, E., Gomez-Pineda, A., Ugazio, A. G., Notarangelo, L. D., et al. (2002) Bone Marrow Transplant. 29, 625-628.
7. Muenchen, M. O. & Barcena, A. (2004) Cancer Control 11, 105-118.
8. Touraine, J. L., Raudrant, D., Golfier, F., Rebaud, A., Sembeil, R., Roncarolo, M. G., Bacchetta, R., D'Oiron, R., Lambert, T. & Gebuhrer, L. (2004) Fetal Diagn. Ther. 19, 305-312.
9. Flake, A. W. & Zanjani, E. D. (1999) Blood 94, 2179-2191.
10. Caillat-Zucman, S., Le Deist, F., Haddad, E., Gannage, M., Dal Cortivo, L., Jabado, N., Hacein-Bey-Abina, S., Blanche, S., Casanova, J. L., Fischer, A., et al. (2004) Bone Marrow Transplant. 33, 1089-1095.
11. Hayashi, S., Peranteau, W. H., Shaaban, A. F. & Flake, A. W. (2002) Blood 100, 804-812.
12. Peranteau, W. H., Hayashi, S., Hsieh, M., Shaaban, A. F. & Flake, A. W. (2002) Blood 100, 2225-2234.
13. Zanjani, E. D. & Anderson, W. F. (1999) Science 285, 2084-2088.
14. Frattini, A., Orchard, P. J., Sobacchi, C., Giliani, S., Abinun, M., Mattsson, J. P., Keeling, D. J., Andersson, A. K., Wallbrandt, P., Zecca, L., et al. (2000) Nat. Genet. 25, 343-346.
15. Kornak, U., Schulz, A., Friedrich, W., Uhlhaas, S., Kremens, B., Voit, T., Hasan, C., Bode, U., Jentsch, T. J. & Kubisch, C. (2000) Hum. Mol. Genet. 9, 2059-2063.
16. Sobacchi, C., Frattini, A., Orchard, P., Porras, O., Tezcan, I., Andolina, M., Babul-Hirji, R., Baric, I., Canham, N., Chitayat, D., et al. (2001) Hum. Mol. Genet. 10, 1767-1773.
17. Frattini, A., Pangrazio, A., Susani, L., Sobacchi, C., Mirolo, M., Abinun, M., Andolina, M., Flanagan, A., Horwitz, E. M., Mihci, E., et al. (2003) J. Bone Miner. Res. 18, 1740-1747.
18. Gerritsen, E. J., Vossen, J. M., Fasth, A., Friedrich, W., Morgan, G., Padmos, A., Vellodi, A., Porras, O., O'Meara, A., Porta, F., et al. (1994) J. Pediatr. 125, 896-902.
19. Eapen, M., Davies, S. M., Ramsay, N. K & Orchard, P. J. (1998) Bone Marrow Transplant. 22, 941-946.
20. Kapelushnik, J., Shalev, C., Yaniv, I., Aker, M., Carmi, R., Cohen, Z., Mozer, A., Schulman, C., Stein, G. & Or, R. (2001) Bone Marrow Transplant. 27, 129-132.
21. Schulz, A. S., Classen, C. F., Mihatsch, W. A., Sigl-Kraetzig, M., Wiesneth, M., Debatin, K. M., Friedrich, W. & Muller, S. M. (2002) Blood 99, 3458-3460.
22. Driessen, G. J., Gerritsen, E. J., Fischer, A., Fasth, A., Hop, W. C., Veys, P., Porta, F., Cant, A., Steward, C. G., Vossen, J. M., et al. (2003) Bone Marrow Transplant. 32, 657-663.
23. Alvarez-Dolado M., Pardal, R., Garcia-Verdugo, J. M., Fike, J. R., Lee, H. O., Pfeffer, K., Lois, C., Morrison, S. J. & Alvarez-Buylla, A. (2003) Nature 425, 968-973.
24. Vassilopoulos, G., Wang, P. R. & Russell, D. W. (2003) Nature 422, 901-904.
25. Camargo, F. D., Finegold, M. & Goodell, M. A. (2004) J. Clin. Invest. 113, 1266-1270.
26. Okabe, M., Ikawa, M., Kominami, K., Nakanishi, T. & Nishimune, Y. (1997) FEBS Lett. 407, 313-319.
27. Blair, H. C., Borysenko, C. V., Villa, A., Schlesinger, P. H., Kalla, S. E., Yaroslavskiy, B. B., Garcia-Palacios, V., Oakley, J. I. & Orchard, P. J. (2004) J. Bone Miner. Res. 19, 1329-1338.
28. Magli, M. C., Dick, J. E., Huszar, D., Bernstein, A. & Phillips, R. A. (1987) Proc. Natl. Acad. Sci. USA 84, 789-793.
29. Cairns, L. A., Moroni, E., Levantini, E., Giorgetti, A., Klinger, F. G., Ronzoni, S., Tatangelo, L., Tiveron, C., De Felici, M., Dolci, S., et al. (2003) Blood 102, 3954-3962.
30. Scimeca, J. C., Franchi, A., Trojani, C., Parrinello, H., Grosgeorge, J., Robert, C., Jaillon, O., Poirier, C., Gaudray, P. & Carle, G. F. (2000) Bone 26, 207-213.
31. Blair, H. C., Teitelbaum, S. L., Ghiselli, R. & Gluck, S. (1989) Science 245, 855-857.
32. Taranta, A., Migliaccio, S., Recchia, I., Caniglia, M., Luciani, M., De Rossi, G., Dionisi-Vici, C., Pinto, R. M., Francalanci, P., Boldrini, R., et al. (2003) Am. J. Pathol. 162, 57-68.
33. Susani, L., Pangrazio, A., Sobacchi, C., Taranta, A., Mortier, G., Savarirayan, R., Villa, A., Orchard, P., Vezzoni, P., Albertini, A., et al. (2004) Hum. Mutat. 24, 225-235.
34. Sykes, M. (2001) Immunity 14, 417-424.
35. Blakemore, K., Hattenburg, C., Stellen, G., Berg, K., South, S., Murphy, K. & Jones, R. (2004) Am. J. Obstet. Gynecol. 190, 960-973.
36. Li, Y. P., Chen, W., Liang, Y., Li, E. & Stashenko, P. (1999) Nat. Genet. 23, 447-451.
37. Anderson, J. M. (2000) Curr. Opin. Hematol. 7, 40-47.