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Results: 1 to 20 of 45

1.

Gene therapy for ADA-SCID: defining the factors for successful outcome.

Gaspar HB.

Blood. 2012 Nov 1;120(18):3628-9. doi: 10.1182/blood-2012-08-446559. No abstract available.

PMID:
23118212
[PubMed - indexed for MEDLINE]
Free Article
2.

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
[PubMed - indexed for MEDLINE]
3.

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.

PMID:
23566838
[PubMed - indexed for MEDLINE]
Free PMC Article
4.

Gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency.

Montiel-Equihua CA, Thrasher AJ, Gaspar HB.

Curr Gene Ther. 2012 Feb 1;12(1):57-65. Review.

PMID:
22348551
[PubMed - indexed for MEDLINE]
5.

Management options for adenosine deaminase deficiency; proceedings of the EBMT satellite workshop (Hamburg, March 2006).

Booth C, Hershfield M, Notarangelo L, Buckley R, Hoenig M, Mahlaoui N, Cavazzana-Calvo M, Aiuti A, Gaspar HB.

Clin Immunol. 2007 May;123(2):139-47. Epub 2007 Feb 14. Review.

PMID:
17300989
[PubMed - indexed for MEDLINE]
6.

Gene therapy of primary T cell immunodeficiencies.

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

Gene. 2013 Aug 10;525(2):170-3. doi: 10.1016/j.gene.2013.03.092. Epub 2013 Apr 10. Review.

PMID:
23583799
[PubMed - indexed for MEDLINE]
7.

Bone marrow transplantation and alternatives for adenosine deaminase deficiency.

Gaspar HB.

Immunol Allergy Clin North Am. 2010 May;30(2):221-36. doi: 10.1016/j.iac.2010.01.002. Review.

PMID:
20493398
[PubMed - indexed for MEDLINE]
8.

Gene transfer to hematopoietic cells--the clinical experience.

Richter J.

Eur J Haematol. 1997 Aug;59(2):67-75. Review. No abstract available.

PMID:
9293853
[PubMed - indexed for MEDLINE]
9.

Towards a rAAV-based gene therapy for ADA-SCID: from ADA deficiency to current and future treatment strategies.

Silver JN, Flotte TR.

Pharmacogenomics. 2008 Jul;9(7):947-68. doi: 10.2217/14622416.9.7.947. Review.

PMID:
18597656
[PubMed - indexed for MEDLINE]
10.

Gene therapy for adenosine-deaminase-deficient severe combined immunodeficiency.

Aiuti A.

Best Pract Res Clin Haematol. 2004 Sep;17(3):505-16. Review.

PMID:
15498720
[PubMed - indexed for MEDLINE]
11.

Gene therapy for adenosine deaminase deficiency.

Cappelli B, Aiuti A.

Immunol Allergy Clin North Am. 2010 May;30(2):249-60. doi: 10.1016/j.iac.2010.02.003. Review.

PMID:
20493400
[PubMed - indexed for MEDLINE]
12.

[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
[PubMed - indexed for MEDLINE]
13.

Bone marrow gene therapy for adenosine deaminase deficiency.

Kaptein LC, Einerhand MP, Braakman E, Valerio D, van Beusechem VW.

Immunodeficiency. 1993;4(1-4):335-45. Review.

PMID:
7909479
[PubMed - indexed for MEDLINE]
14.

Primary immunodeficiencies: molecular aspects and treatment.

Fischer A.

Bone Marrow Transplant. 1992;9 Suppl 1:39-43. Review. No abstract available.

PMID:
1354525
[PubMed - indexed for MEDLINE]
15.

Gene therapy for severe combined immunodeficiency caused by adenosine deaminase deficiency: improved retroviral vectors for clinical trials.

Onodera M, Nelson DM, Sakiyama Y, Candotti F, Blaese RM.

Acta Haematol. 1999;101(2):89-96. Review.

PMID:
10202239
[PubMed - indexed for MEDLINE]
16.

Gene therapy for severe combined immunodeficiency: are we there yet?

Cavazzana-Calvo M, Fischer A.

J Clin Invest. 2007 Jun;117(6):1456-65. Review.

PMID:
17549248
[PubMed - indexed for MEDLINE]
Free PMC Article
17.

Advances in gene therapy for ADA-deficient SCID.

Aiuti A.

Curr Opin Mol Ther. 2002 Oct;4(5):515-22. Review.

PMID:
12435054
[PubMed - indexed for MEDLINE]
18.

Adenosine deaminase deficiency as the first target disorder in gene therapy.

Onodera M, Sakiyama Y.

Expert Opin Investig Drugs. 2000 Mar;9(3):543-9. Review.

PMID:
11060694
[PubMed - indexed for MEDLINE]
19.

Gene therapy for primary immunodeficiencies: Part 1.

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

Curr Opin Immunol. 2012 Oct;24(5):580-4. doi: 10.1016/j.coi.2012.08.008. Epub 2012 Sep 12. Review.

PMID:
22981681
[PubMed - indexed for MEDLINE]
20.

Development of gene therapy for immunodeficiency: adenosine deaminase deficiency.

Blaese RM.

Pediatr Res. 1993 Jan;33(1 Suppl):S49-53; discussion S53-5. Review.

PMID:
8433875
[PubMed - indexed for MEDLINE]

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