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Results: 18

1.

Hematopoietic stem cell gene therapy:assessing the relevance of preclinical models.

Larochelle A, Dunbar CE.

Semin Hematol. 2013 Apr;50(2):101-30. Review. No abstract available.

PMID:
24014892
[PubMed - indexed for MEDLINE]
Free PMC Article
2.

Comparison of insulators and promoters for expression of the Wiskott-Aldrich syndrome protein using lentiviral vectors.

Koldej RM, Carney G, Wielgosz MM, Zhou S, Zhan J, Sorrentino BP, Nienhuis AW.

Hum Gene Ther Clin Dev. 2013 Jun;24(2):77-85. doi: 10.1089/humc.2012.244. Epub 2013 Jun 20.

PMID:
23786330
[PubMed - indexed for MEDLINE]
Free PMC Article
3.

The genetic basis of severe combined immunodeficiency and its variants.

Tasher D, Dalal I.

Appl Clin Genet. 2012 Aug 7;5:67-80. doi: 10.2147/TACG.S18693. Print 2012.

PMID:
23776382
[PubMed]
Free PMC Article
4.

Mouse transplant models for evaluating the oncogenic risk of a self-inactivating XSCID lentiviral vector.

Zhou S, Ma Z, Lu T, Janke L, Gray JT, Sorrentino BP.

PLoS One. 2013 Apr 23;8(4):e62333. doi: 10.1371/journal.pone.0062333. Print 2013.

PMID:
23626802
[PubMed - indexed for MEDLINE]
Free PMC Article
5.

Biosafety features of lentiviral vectors.

Schambach A, Zychlinski D, Ehrnstroem B, Baum C.

Hum Gene Ther. 2013 Feb;24(2):132-42. doi: 10.1089/hum.2012.229. Review.

PMID:
23311447
[PubMed - indexed for MEDLINE]
Free PMC Article
6.
7.

Alpharetroviral vector-mediated gene therapy for X-CGD: functional correction and lack of aberrant splicing.

Kaufmann KB, Brendel C, Suerth JD, Mueller-Kuller U, Chen-Wichmann L, Schwäble J, Pahujani S, Kunkel H, Schambach A, Baum C, Grez M.

Mol Ther. 2013 Mar;21(3):648-61. doi: 10.1038/mt.2012.249. Epub 2012 Dec 4.

PMID:
23207695
[PubMed - indexed for MEDLINE]
Free PMC Article
8.

Transduction of human CD34+ repopulating cells with a self-inactivating lentiviral vector for SCID-X1 produced at clinical scale by a stable cell line.

Greene MR, Lockey T, Mehta PK, Kim YS, Eldridge PW, Gray JT, Sorrentino BP.

Hum Gene Ther Methods. 2012 Oct;23(5):297-308. doi: 10.1089/hgtb.2012.150. Epub 2012 Nov 7.

PMID:
23075105
[PubMed - indexed for MEDLINE]
Free PMC Article
9.

Thymidine kinase suicide gene-mediated ganciclovir ablation of autologous gene-modified rhesus hematopoiesis.

Barese CN, Krouse AE, Metzger ME, King CA, Traversari C, Marini FC, Donahue RE, Dunbar CE.

Mol Ther. 2012 Oct;20(10):1932-43. doi: 10.1038/mt.2012.166. Epub 2012 Aug 21.

PMID:
22910293
[PubMed - indexed for MEDLINE]
Free PMC Article
10.

Correction of murine Rag2 severe combined immunodeficiency by lentiviral gene therapy using a codon-optimized RAG2 therapeutic transgene.

van Til NP, de Boer H, Mashamba N, Wabik A, Huston M, Visser TP, Fontana E, Poliani PL, Cassani B, Zhang F, Thrasher AJ, Villa A, Wagemaker G.

Mol Ther. 2012 Oct;20(10):1968-80. doi: 10.1038/mt.2012.110. Epub 2012 Jun 12.

PMID:
22692499
[PubMed - indexed for MEDLINE]
Free PMC Article
11.

Hematopoietic-stem-cell-based gene therapy for HIV disease.

Kiem HP, Jerome KR, Deeks SG, McCune JM.

Cell Stem Cell. 2012 Feb 3;10(2):137-47. doi: 10.1016/j.stem.2011.12.015. Review.

PMID:
22305563
[PubMed - indexed for MEDLINE]
Free PMC Article
12.

Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning.

Huston MW, van Til NP, Visser TP, Arshad S, Brugman MH, Cattoglio C, Nowrouzi A, Li Y, Schambach A, Schmidt M, Baum C, von Kalle C, Mavilio F, Zhang F, Blundell MP, Thrasher AJ, Verstegen MM, Wagemaker G.

Mol Ther. 2011 Oct;19(10):1867-77. doi: 10.1038/mt.2011.127. Epub 2011 Jul 12.

PMID:
21750532
[PubMed - indexed for MEDLINE]
Free PMC Article
13.

Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease-mediated safe harbor targeting.

Zou J, Sweeney CL, Chou BK, Choi U, Pan J, Wang H, Dowey SN, Cheng L, Malech HL.

Blood. 2011 May 26;117(21):5561-72. doi: 10.1182/blood-2010-12-328161. Epub 2011 Mar 16.

PMID:
21411759
[PubMed - indexed for MEDLINE]
Free PMC Article
14.

Reconstitutable charged polymeric (PLGA)(2)-b-PEI micelles for gene therapeutics delivery.

Mishra D, Kang HC, Bae YH.

Biomaterials. 2011 May;32(15):3845-54. doi: 10.1016/j.biomaterials.2011.01.077. Epub 2011 Feb 26.

PMID:
21354616
[PubMed - indexed for MEDLINE]
Free PMC Article
15.

The use of chromatin insulators to improve the expression and safety of integrating gene transfer vectors.

Emery DW.

Hum Gene Ther. 2011 Jun;22(6):761-74. doi: 10.1089/hum.2010.233. Epub 2011 Mar 25. Review.

PMID:
21247248
[PubMed - indexed for MEDLINE]
Free PMC Article
16.

Chronic granulomatous disease: lessons from a rare disorder.

Segal BH, Veys P, Malech H, Cowan MJ.

Biol Blood Marrow Transplant. 2011 Jan;17(1 Suppl):S123-31. doi: 10.1016/j.bbmt.2010.09.008. Review.

PMID:
21195301
[PubMed - indexed for MEDLINE]
Free PMC Article
17.

Meganucleases and other tools for targeted genome engineering: perspectives and challenges for gene therapy.

Silva G, Poirot L, Galetto R, Smith J, Montoya G, Duchateau P, Pâques F.

Curr Gene Ther. 2011 Feb;11(1):11-27. Review.

PMID:
21182466
[PubMed - indexed for MEDLINE]
Free PMC Article
18.

Genetics of SCID.

Cossu F.

Ital J Pediatr. 2010 Nov 15;36:76. doi: 10.1186/1824-7288-36-76. Review.

PMID:
21078154
[PubMed - indexed for MEDLINE]
Free PMC Article

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