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Items: 1 to 20 of 25

1.

Efficacy of gene therapy for X-linked severe combined immunodeficiency.

Hacein-Bey-Abina S, Hauer J, Lim A, Picard C, Wang GP, Berry CC, Martinache C, Rieux-Laucat F, Latour S, Belohradsky BH, Leiva L, Sorensen R, Debré M, Casanova JL, Blanche S, Durandy A, Bushman FD, Fischer A, Cavazzana-Calvo M.

N Engl J Med. 2010 Jul 22;363(4):355-64. doi: 10.1056/NEJMoa1000164.

2.

[Transplantation of genetically modified cells in the treatment of children with SCID: great hopes and recent disappointments].

Smogorzewska EM, Weinberg KI, Kohn DB.

Med Wieku Rozwoj. 2003 Jan-Mar;7(1):27-34. Review. Polish.

PMID:
13130167
3.

[Gene therapy of X-linked severe combined immunologic deficiency (SCID-X1)].

Hacein-Bey S, Gross F, Nusbaum P, Yvon E, Fischer A, Cavazzana-Calvo M.

Pathol Biol (Paris). 2001 Feb;49(1):57-66. Review. French.

PMID:
11265225
4.

Gene therapy for severe combined immunodeficiencies.

Gaspar HB, Thrasher AJ.

Expert Opin Biol Ther. 2005 Sep;5(9):1175-82. Review.

PMID:
16120048
5.

Gene therapy of X-linked severe combined immunodeficiency.

Hacein-Bey-Abina S, Fischer A, Cavazzana-Calvo M.

Int J Hematol. 2002 Nov;76(4):295-8. Review.

PMID:
12463590
6.

Advances in the understanding and treatment of human severe combined immunodeficiency.

Buckley RH.

Immunol Res. 2000;22(2-3):237-51. Review.

PMID:
11339359
7.

Gene therapy for PIDs: progress, pitfalls and prospects.

Mukherjee S, Thrasher AJ.

Gene. 2013 Aug 10;525(2):174-81. doi: 10.1016/j.gene.2013.03.098. Epub 2013 Apr 6. Review.

8.

Relationship between patterns of engraftment in peripheral blood and immune reconstitution after allogeneic bone marrow transplantation for (severe) combined immunodeficiency.

van Leeuwen JE, van Tol MJ, Joosten AM, Schellekens PT, van den Bergh RL, Waaijer JL, Oudeman-Gruber NJ, van der Weijden-Ragas CP, Roos MT, Gerritsen EJ, et al.

Blood. 1994 Dec 1;84(11):3936-47. Review.

9.

Haploidentical bone marrow stem cell transplantation in human severe combined immunodeficiency.

Buckley RH, Schiff SE, Schiff RI, Roberts JL, Markert ML, Peters W, Williams LW, Ward FE.

Semin Hematol. 1993 Oct;30(4 Suppl 4):92-101; discussion 102-4. Review.

PMID:
7905667
10.

Perspectives of gene therapy for primary immunodeficiencies.

Chinen J, Puck JM.

Curr Opin Allergy Clin Immunol. 2004 Dec;4(6):523-7. Review.

PMID:
15640693
11.

Gene therapy in infants with severe combined immunodeficiency.

Otsu M, Candotti F.

BioDrugs. 2002;16(4):229-39. Review.

PMID:
12196037
12.

B-cell reconstitution for SCID: should a conditioning regimen be used in SCID treatment?

Haddad E, Leroy S, Buckley RH.

J Allergy Clin Immunol. 2013 Apr;131(4):994-1000. doi: 10.1016/j.jaci.2013.01.047. Epub 2013 Mar 5. Review.

13.

Severe combined immunodeficiency (SCID): from molecular basis to clinical management.

Sponzilli I, Notarangelo LD.

Acta Biomed. 2011 Apr;82(1):5-13. Review.

PMID:
22069950
14.

Severe combined immunodeficiency. A model disease for molecular immunology and therapy.

Fischer A, Le Deist F, Hacein-Bey-Abina S, André-Schmutz I, Basile Gde S, de Villartay JP, Cavazzana-Calvo M.

Immunol Rev. 2005 Feb;203:98-109. Review.

PMID:
15661024
15.

Transplantation of hematopoietic stem cells in human severe combined immunodeficiency: longterm outcomes.

Buckley RH.

Immunol Res. 2011 Apr;49(1-3):25-43. doi: 10.1007/s12026-010-8191-9. Review.

16.

Update on gene therapy for adenosine deaminase-deficient severe combined immunodeficiency.

Ferrua F, Brigida I, Aiuti A.

Curr Opin Allergy Clin Immunol. 2010 Dec;10(6):551-6. doi: 10.1097/ACI.0b013e32833fea85. Review.

PMID:
20966749
17.

Gene therapy for inherited immunodeficiency.

Touzot F, Hacein-Bey-Abina S, Fischer A, Cavazzana M.

Expert Opin Biol Ther. 2014 Jun;14(6):789-98. doi: 10.1517/14712598.2014.895811. Epub 2014 Mar 8. Review.

PMID:
24823313
18.
19.

B-cell function in severe combined immunodeficiency after stem cell or gene therapy: a review.

Buckley RH.

J Allergy Clin Immunol. 2010 Apr;125(4):790-7. doi: 10.1016/j.jaci.2010.02.012. Review.

20.

Gene therapy for primary immunodeficiencies: current status and future prospects.

Qasim W, Gennery AR.

Drugs. 2014 Jun;74(9):963-9. doi: 10.1007/s40265-014-0223-7. Review.

PMID:
24848753
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