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Items: 1 to 20 of 197

1.

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
2.

Early determinants of long-term T-cell reconstitution after hematopoietic stem cell transplantation for severe combined immunodeficiency.

Borghans JA, Bredius RG, Hazenberg MD, Roelofs H, Jol-van der Zijde EC, Heidt J, Otto SA, Kuijpers TW, Fibbe WE, Vossen JM, Miedema F, van Tol MJ.

Blood. 2006 Jul 15;108(2):763-9.

3.

Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning.

Gaspar HB, Bjorkegren E, Parsley K, Gilmour KC, King D, Sinclair J, Zhang F, Giannakopoulos A, Adams S, Fairbanks LD, Gaspar J, Henderson L, Xu-Bayford JH, Davies EG, Veys PA, Kinnon C, Thrasher AJ.

Mol Ther. 2006 Oct;14(4):505-13. Epub 2006 Aug 14.

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.

Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy.

Hacein-Bey-Abina S, Le Deist F, Carlier F, Bouneaud C, Hue C, De Villartay JP, Thrasher AJ, Wulffraat N, Sorensen R, Dupuis-Girod S, Fischer A, Davies EG, Kuis W, Leiva L, Cavazzana-Calvo M.

N Engl J Med. 2002 Apr 18;346(16):1185-93.

6.

Gene therapy of severe combined immunodeficiencies.

Cavazzana-Calvo M, Hacein-Bey S, Yates F, de Villartay JP, Le Deist F, Fischer A.

J Gene Med. 2001 May-Jun;3(3):201-6. Review.

PMID:
11437325
7.

Hematopoietic stem cell transplantation for severe combined immune deficiency or what the children have taught us.

Rappeport JM, O'Reilly RJ, Kapoor N, Parkman R.

Immunol Allergy Clin North Am. 2010 Feb;30(1):17-30. doi: 10.1016/j.iac.2009.10.002. Review.

PMID:
20113884
8.

Competition within the early B-cell compartment conditions B-cell reconstitution after hematopoietic stem cell transplantation in nonirradiated recipients.

Liu A, Vosshenrich CA, Lagresle-Peyrou C, Malassis-Seris M, Hue C, Fischer A, Di Santo JP, Cavazzana-Calvo M.

Blood. 2006 Aug 15;108(4):1123-8. Epub 2006 Apr 13.

9.

Clonal evidence for the transduction of CD34+ cells with lymphomyeloid differentiation potential and self-renewal capacity in the SCID-X1 gene therapy trial.

Schmidt M, Hacein-Bey-Abina S, Wissler M, Carlier F, Lim A, Prinz C, Glimm H, Andre-Schmutz I, Hue C, Garrigue A, Le Deist F, Lagresle C, Fischer A, Cavazzana-Calvo M, von Kalle C.

Blood. 2005 Apr 1;105(7):2699-706. Epub 2004 Dec 7.

10.

Hematopoietic stem cell transplantation for severe combined immune deficiency.

Weinberg KI, Kapoor N, Shah AJ, Crooks GM, Kohn DB, Parkman R.

Curr Allergy Asthma Rep. 2001 Sep;1(5):416-20. Review.

PMID:
11892067
11.

Hematopoietic stem cell transplantation for severe combined immunodeficiency diseases.

Cowan MJ, Neven B, Cavazanna-Calvo M, Fischer A, Puck J.

Biol Blood Marrow Transplant. 2008 Jan;14(1 Suppl 1):73-5. doi: 10.1016/j.bbmt.2007.10.017. Review. Erratum in: Biol Blood Marrow Transplant. 2008 Nov;14(11):1317-8.

12.

Treatment of an infant with X-linked severe combined immunodeficiency (SCID-X1) by gene therapy in Australia.

Ginn SL, Curtin JA, Kramer B, Smyth CM, Wong M, Kakakios A, McCowage GB, Watson D, Alexander SI, Latham M, Cunningham SC, Zheng M, Hobson L, Rowe PB, Fischer A, Cavazzana-Calvo M, Hacein-Bey-Abina S, Alexander IE.

Med J Aust. 2005 May 2;182(9):458-63.

PMID:
15865589
13.

[Gene therapy of severe combined immunodeficiency disease: proof of principle of efficiency and safety issues. Gene therapy, primary immunodeficiencies, retrovirus, lentivirus, genome].

Fischer A, Hacein-Bey-Abina S, Lagresle C, Garrigue A, Cavazana-Calvo M.

Bull Acad Natl Med. 2005 May;189(5):779-85; discussion 786-8. French.

PMID:
16433450
14.

[Gene therapy for hereditary immunodeficiencies].

Fischer A, de Saint-Basile G, Disanto JP, Hacein-Bey S, Sharara L, Cavazzana-Calvo M.

C R Seances Soc Biol Fil. 1996;190(1):77-93. Review. French.

PMID:
8881271
15.

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
16.

Gene therapy in infants with severe combined immunodeficiency.

Otsu M, Candotti F.

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

PMID:
12196037
17.

In vivo correction of ZAP-70 immunodeficiency by intrathymic gene transfer.

Adjali O, Marodon G, Steinberg M, Mongellaz C, Thomas-Vaslin V, Jacquet C, Taylor N, Klatzmann D.

J Clin Invest. 2005 Aug;115(8):2287-95.

18.

Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease.

Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, Gross F, Yvon E, Nusbaum P, Selz F, Hue C, Certain S, Casanova JL, Bousso P, Deist FL, Fischer A.

Science. 2000 Apr 28;288(5466):669-72.

19.
20.

Neonatal bone marrow transplantation of ADA-deficient SCID mice results in immunologic reconstitution despite low levels of engraftment and an absence of selective donor T lymphoid expansion.

Carbonaro DA, Jin X, Cotoi D, Mi T, Yu XJ, Skelton DC, Dorey F, Kellems RE, Blackburn MR, Kohn DB.

Blood. 2008 Jun 15;111(12):5745-54. doi: 10.1182/blood-2007-08-103663. Epub 2008 Mar 20.

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