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Results: 1 to 20 of 142

1.

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]
2.

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
3.

Ten years of gene therapy for primary immune deficiencies.

Aiuti A, Roncarolo MG.

Hematology Am Soc Hematol Educ Program. 2009:682-9. doi: 10.1182/asheducation-2009.1.682. Review.

PMID:
20008254
[PubMed - indexed for MEDLINE]
Free Article
4.

Strategies for retrovirus-based correction of severe, combined immunodeficiency (SCID).

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

Methods Enzymol. 2012;507:15-27. doi: 10.1016/B978-0-12-386509-0.00002-8.

PMID:
22365767
[PubMed - indexed for MEDLINE]
5.

Gene therapy for primary adaptive immune deficiencies.

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

J Allergy Clin Immunol. 2011 Jun;127(6):1356-9. doi: 10.1016/j.jaci.2011.04.030. Review.

PMID:
21624615
[PubMed - indexed for MEDLINE]
6.

Parachuting in the epigenome: the biology of gene vector insertion profiles in the context of clinical trials.

Baum C.

EMBO Mol Med. 2011 Feb;3(2):75-7. doi: 10.1002/emmm.201000110. Epub 2011 Jan 21.

PMID:
21254403
[PubMed - indexed for MEDLINE]
Free PMC Article
7.

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]
8.

[Current status and future prospects of stem cell gene therapy for primary immunodeficiency].

Uchiyama T, Onodera M.

Nihon Rinsho Meneki Gakkai Kaishi. 2013;36(3):148-55. Review. Japanese.

PMID:
23812072
[PubMed - indexed for MEDLINE]
Free Article
9.

Progress and prospects: gene therapy for inherited immunodeficiencies.

Qasim W, Gaspar HB, Thrasher AJ.

Gene Ther. 2009 Nov;16(11):1285-91. doi: 10.1038/gt.2009.127. Epub 2009 Sep 24. Review.

PMID:
19776764
[PubMed - indexed for MEDLINE]
10.

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.

PMID:
24823313
[PubMed - in process]
11.

Gene therapy of inherited immunodeficiencies.

Santilli G, Thornhill SI, Kinnon C, Thrasher AJ.

Expert Opin Biol Ther. 2008 Apr;8(4):397-407. doi: 10.1517/14712598.8.4.397 . Review.

PMID:
18352845
[PubMed - indexed for MEDLINE]
12.

Gene therapy for severe combined immunodeficiencies.

Gaspar HB, Thrasher AJ.

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

PMID:
16120048
[PubMed - indexed for MEDLINE]
13.

Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction.

Gaspar HB, Cooray S, Gilmour KC, Parsley KL, Zhang F, Adams S, Bjorkegren E, Bayford J, Brown L, Davies EG, Veys P, Fairbanks L, Bordon V, Petropoulou T, Kinnon C, Thrasher AJ.

Sci Transl Med. 2011 Aug 24;3(97):97ra80. doi: 10.1126/scitranslmed.3002716. Erratum in: Sci Transl Med. 2013 Jan 16;5(168):168er1. Petropolou, Theoni [corrected to Petropoulou, Theoni].

PMID:
21865538
[PubMed - indexed for MEDLINE]
Free Article
14.

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]
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.

[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]
17.

Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy.

Cassani B, Montini E, Maruggi G, Ambrosi A, Mirolo M, Selleri S, Biral E, Frugnoli I, Hernandez-Trujillo V, Di Serio C, Roncarolo MG, Naldini L, Mavilio F, Aiuti A.

Blood. 2009 Oct 22;114(17):3546-56. doi: 10.1182/blood-2009-02-202085. Epub 2009 Aug 3.

PMID:
19652199
[PubMed - indexed for MEDLINE]
Free Article
18.

Development of novel efficient SIN vectors with improved safety features for Wiskott-Aldrich syndrome stem cell based gene therapy.

Avedillo Díez I, Zychlinski D, Coci EG, Galla M, Modlich U, Dewey RA, Schwarzer A, Maetzig T, Mpofu N, Jaeckel E, Boztug K, Baum C, Klein C, Schambach A.

Mol Pharm. 2011 Oct 3;8(5):1525-37. doi: 10.1021/mp200132u. Epub 2011 Aug 31.

PMID:
21851067
[PubMed - indexed for MEDLINE]
19.

Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID.

Aiuti A, Brigida I, Ferrua F, Cappelli B, Chiesa R, Marktel S, Roncarolo MG.

Immunol Res. 2009;44(1-3):150-9. doi: 10.1007/s12026-009-8107-8.

PMID:
19224139
[PubMed - indexed for MEDLINE]
20.

Gene therapy for primary immunodeficiencies: Part 2.

Aiuti A, Bacchetta R, Seger R, Villa A, Cavazzana-Calvo M.

Curr Opin Immunol. 2012 Oct;24(5):585-91. doi: 10.1016/j.coi.2012.07.012. Epub 2012 Aug 18. Review.

PMID:
22909900
[PubMed - indexed for MEDLINE]

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