1 |
Pasvolsky R, Feigelson SW, Kilic SS, Simon AJ, Tal-Lapidot G, Grabovsky V, et al. A LAD-III syndrome is associated with defective expression of the Rap-1 activator CalDAG- GEFI in lymphocytes, neutrophils, and platelets. J Exp Med 2007;204:1571-82
DOI
PUBMED
|
2 |
Buckley RH, Schiff SE, Schiff RI, Markert L, Williams LW, Roberts JL, et al. Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency. N Engl J Med 1999;340:508-16
DOI
ScienceOn
|
3 |
Fischer A, Landais P, Friedrich W, Morgan G, Gerritsen B, Fasth A, et al. European experience of bone-marrow transplantation for severe combined immunodeficiency. Lancet 1990;336:850-4
DOI
ScienceOn
|
4 |
Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, Gross F, Yvon E, Nusbaum P, et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 2000;288:669-72
DOI
PUBMED
|
5 |
Ochs HD, Notarangelo LD. Structure and function of the Wiskott-Aldrich syndrome protein. Curr Opin Hematol 2005; 12:284-91
DOI
ScienceOn
|
6 |
Filipovich AH, Stone JV, Tomany SC, Ireland M, Kollman C, Pelz CJ, et al. Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program. Blood 2001;97:1598-603
DOI
ScienceOn
|
7 |
Bauer TR, Jr., Hai M, Tuschong LM, Burkholder TH, Gu YC, Sokolic RA, et al. Correction of the disease phenotype in canine leukocyte adhesion deficiency using ex vivo hematopoietic stem cell gene therapy. Blood 2006;108:3313-20
DOI
ScienceOn
|
8 |
Roos D, de Boer M, Kuribayashi F, Meischl C, Weening RS, Segal AW, et al. Mutations in the X-linked and autosomal recessive forms of chronic granulomatous disease. Blood 1996;87:1663-81
PUBMED
ScienceOn
|
9 |
Hacein-Bey-Abina S, von Kalle C, Schmidt M, Le Deist F, Wulffraat N, McIntyre E, et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2003;348:255-6
DOI
ScienceOn
|
10 |
Malech HL, Maples PB, Whiting-Theobald N, Linton GF, Sekhsaria S, Vowells SJ, et al. Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease. Proc Natl Acad Sci U S A 1997;94:12133-8
DOI
ScienceOn
|
11 |
Kang HJ, Shin HY, Ko SH, Park JA, Kim EK, Rhim JW, et al. Unrelated bone marrow transplantation with a reduced toxicity myeloablative conditioning regimen in Wiskott-Aldrich syndrome. J Korean Med Sci 2008;23:146-8
DOI
ScienceOn
|
12 |
Gatti RA, Meuwissen HJ, Allen HD, Hong R, Good RA. Immunological reconstitution of sex-linked lymphopenic immunological deficiency. Lancet 1968;2:1366-9
PUBMED
|
13 |
Keightley RG, Lawton AR, Cooper MD, Yunis EJ. Successful fetal liver transplantation in a child with severe combined immunodeficiency. Lancet 1975;2:850-3
PUBMED
|
14 |
Marodi L, Notarangelo LD. Immunological and genetic bases of new primary immunodeficiencies. Nat Rev Immunol 2007; 7:851-61
DOI
ScienceOn
|
15 |
Fischer R. Allogeneic hematopietic stem cell transplantation for congenital immune deficiencies. In Atkinson K, Champlin R, Ritz J, Fibbe WE, Ljungman P, Brenner MK, eds. Clinical bone marrow and Blood Stem Cell Transplantation. Cambridge: Cambridge University Press 2004;947-61
|
16 |
Roesler J, Brenner S, Bukovsky AA, Whiting-Theobald N, Dull T, Kelly M, et al. Third-generation, self-inactivating gp91(phox) lentivector corrects the oxidase defect in NOD/ SCID mouse-repopulating peripheral blood-mobilized CD34+ cells from patients with X-linked chronic granulomatous disease. Blood 2002;100:4381-90
DOI
PUBMED
ScienceOn
|
17 |
Qasim W, Gaspar HB, Thrasher AJ. Gene therapy for severe combined immune deficiency. Expert Rev Mol Med 2004;6: 1-15
PUBMED
|
18 |
Buckley RH, Schiff SE, Schiff RI, Markert L, Williams LW, Roberts JL, et al. Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency. N Engl J Med 1999;340:508-16
DOI
ScienceOn
|
19 |
Small TN, Friedrich W, O'Reilly RJ. Hematopoietic cell transplantation for immune deficiency diseases. In Blume KG, Forman SJ, Appelbaum FR, eds. Thomas' Hematopoietic Cell Transplantation. Massachusetts: Blackwell Publishing Ltd. 2004;:1430-42
|
20 |
Alon R, Aker M, Feigelson S, Sokolovsky-Eisenberg M, Staunton DE, Cinamon G, et al. A novel genetic leukocyte adhesion deficiency in subsecond triggering of integrin avidity by endothelial chemokines results in impaired leukocyte arrest on vascular endothelium under shear flow. Blood 2003;101:4437-45
DOI
ScienceOn
|
21 |
Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U, et al. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med 2006;12: 401-9
DOI
ScienceOn
|
22 |
Rocha V, Wagner JE, Jr., Sobocinski KA, Klein JP, Zhang MJ, Horowitz MM, et al. Graft-versus-host disease in children who have received a cord-blood or bone marrow transplant from an HLA-identical sibling. Eurocord and International Bone Marrow Transplant Registry Working Committee on Alternative Donor and Stem Cell Sources. N Engl J Med 2000;342:1846-54
DOI
ScienceOn
|
23 |
Rubocki RJ, Parsa JR, Hershfield MS, Sanger WG, Pirruccello SJ, Santisteban I, et al. Full hematopoietic engraftment after allogeneic bone marrow transplantation without cytoreduction in a child with severe combined immunodeficiency. Blood 2001;97:809-11
DOI
ScienceOn
|
24 |
Kang EM, Malech HL. Advances in treatment for chronic granulomatous disease. Immunol Res 2009;43:77-84
DOI
ScienceOn
|
25 |
Aiuti A, Slavin S, Aker M, Ficara F, Deola S, Mortellaro A, et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 2002; 296:2410-3
DOI
PUBMED
ScienceOn
|
26 |
Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003;302:415-9
DOI
PUBMED
ScienceOn
|
27 |
Friedrich W, Muller SM. Allogeneic stem cell transplantation for treatment of immunodeficiency. Springer Semin Immunopathol 2004;26:109-18
DOI
ScienceOn
|
28 |
Bach FH, Albertini RJ, Joo P, Anderson JL, Bortin MM. Bone-marrow transplantation in a patient with the Wiskott- Aldrich syndrome. Lancet 1968;2:1364-6
PUBMED
|
29 |
Babior BM. The respiratory burst oxidase and the molecular basis of chronic granulomatous disease. Am J Hematol 1991; 37:263-6
DOI
PUBMED
ScienceOn
|
30 |
Smith RM, Curnutte JT. Molecular basis of chronic granulomatous disease. Blood 1991;77:673-86
PUBMED
ScienceOn
|
31 |
Puck JM, Candotti F. Lessons from the Wiskott-Aldrich syndrome. N Engl J Med 2006;355:1759-61
DOI
ScienceOn
|
32 |
O'Reilly RJ. et al. Fetal liver transplantation in man and animals. In de Gale RP, eds. Recent Advances in Bone Marrow Transplantation. New York: Alan R. Liss. 1989;780-830
|