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Results: 1 to 20 of 44

1.

Restoration of NET formation by gene therapy in CGD controls aspergillosis.

Bianchi M, Hakkim A, Brinkmann V, Siler U, Seger RA, Zychlinsky A, Reichenbach J.

Blood. 2009 Sep 24;114(13):2619-22. doi: 10.1182/blood-2009-05-221606. Epub 2009 Jun 18.

PMID:
19541821
[PubMed - indexed for MEDLINE]
Free PMC Article
2.

Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

Aiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani B, Callegaro L, Scaramuzza S, Andolfi G, Mirolo M, Brigida I, Tabucchi A, Carlucci F, Eibl M, Aker M, Slavin S, Al-Mousa H, Al Ghonaium A, Ferster A, Duppenthaler A, Notarangelo L, Wintergerst U, Buckley RH, Bregni M, Marktel S, Valsecchi MG, Rossi P, Ciceri F, Miniero R, Bordignon C, Roncarolo MG.

N Engl J Med. 2009 Jan 29;360(5):447-58. doi: 10.1056/NEJMoa0805817.

PMID:
19179314
[PubMed - indexed for MEDLINE]
Free Article
3.

High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR).

Schmidt M, Schwarzwaelder K, Bartholomae C, Zaoui K, Ball C, Pilz I, Braun S, Glimm H, von Kalle C.

Nat Methods. 2007 Dec;4(12):1051-7.

PMID:
18049469
[PubMed - indexed for MEDLINE]
4.

An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation.

Ryu BY, Evans-Galea MV, Gray JT, Bodine DM, Persons DA, Nienhuis AW.

Blood. 2008 Feb 15;111(4):1866-75. Epub 2007 Nov 8.

PMID:
17991809
[PubMed - indexed for MEDLINE]
Free PMC Article
5.

Simian immunodeficiency virus lentivector corrects human X-linked chronic granulomatous disease in the NOD/SCID mouse xenograft.

Naumann N, De Ravin SS, Choi U, Moayeri M, Whiting-Theobald N, Linton GF, Ikeda Y, Malech HL.

Gene Ther. 2007 Nov;14(21):1513-24. Epub 2007 Aug 30.

PMID:
17728796
[PubMed - indexed for MEDLINE]
Free PMC Article
6.

Suppression of clonal dominance in cultured human lymphoid cells by addition of the cHS4 insulator to a lentiviral vector.

Evans-Galea MV, Wielgosz MM, Hanawa H, Srivastava DK, Nienhuis AW.

Mol Ther. 2007 Apr;15(4):801-9. Epub 2007 Feb 13.

PMID:
17299406
[PubMed - indexed for MEDLINE]
7.

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.

PMID:
16905365
[PubMed - indexed for MEDLINE]
8.

Unique risk factors for insertional mutagenesis in a mouse model of XSCID gene therapy.

Shou Y, Ma Z, Lu T, Sorrentino BP.

Proc Natl Acad Sci U S A. 2006 Aug 1;103(31):11730-5. Epub 2006 Jul 24.

PMID:
16864781
[PubMed - indexed for MEDLINE]
Free PMC Article
9.

Gene therapy: therapeutic gene causing lymphoma.

Woods NB, Bottero V, Schmidt M, von Kalle C, Verma IM.

Nature. 2006 Apr 27;440(7088):1123.

PMID:
16641981
[PubMed - indexed for MEDLINE]
10.

Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1.

Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U, Glimm H, Kühlcke K, Schilz A, Kunkel H, Naundorf S, Brinkmann A, Deichmann A, Fischer M, Ball C, Pilz I, Dunbar C, Du Y, Jenkins NA, Copeland NG, Lüthi U, Hassan M, Thrasher AJ, Hoelzer D, von Kalle C, Seger R, Grez M.

Nat Med. 2006 Apr;12(4):401-9. Epub 2006 Apr 2.

PMID:
16582916
[PubMed - indexed for MEDLINE]
11.

Effects of busulfan dose escalation on engraftment of infant rhesus monkey hematopoietic stem cells after gene marking by a lentiviral vector.

Kahl CA, Tarantal AF, Lee CI, Jimenez DF, Choi C, Pepper K, Petersen D, Fletcher MD, Leapley AC, Fisher J, Burns TS, Ultsch MN, Dorey FJ, Kohn DB.

Exp Hematol. 2006 Mar;34(3):369-81.

PMID:
16543071
[PubMed - indexed for MEDLINE]
12.

Busulfan pharmacokinetics, toxicity, and low-dose conditioning for autologous transplantation of genetically modified hematopoietic stem cells in the rhesus macaque model.

Kang EM, Hsieh MM, Metzger M, Krouse A, Donahue RE, Sadelain M, Tisdale JF.

Exp Hematol. 2006 Feb;34(2):132-9.

PMID:
16459181
[PubMed - indexed for MEDLINE]
Free PMC Article
13.

Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells.

Calmels B, Ferguson C, Laukkanen MO, Adler R, Faulhaber M, Kim HJ, Sellers S, Hematti P, Schmidt M, von Kalle C, Akagi K, Donahue RE, Dunbar CE.

Blood. 2005 Oct 1;106(7):2530-3. Epub 2005 Jun 2.

PMID:
15933056
[PubMed - indexed for MEDLINE]
Free PMC Article
14.

Distinct genomic integration of MLV and SIV vectors in primate hematopoietic stem and progenitor cells.

Hematti P, Hong BK, Ferguson C, Adler R, Hanawa H, Sellers S, Holt IE, Eckfeldt CE, Sharma Y, Schmidt M, von Kalle C, Persons DA, Billings EM, Verfaillie CM, Nienhuis AW, Wolfsberg TG, Dunbar CE, Calmels B.

PLoS Biol. 2004 Dec;2(12):e423. Epub 2004 Nov 23.

PMID:
15550989
[PubMed - indexed for MEDLINE]
Free PMC Article
15.

Unusual late presentation of X-linked chronic granulomatous disease in an adult female with a somatic mosaic for a novel mutation in CYBB.

Wolach B, Scharf Y, Gavrieli R, de Boer M, Roos D.

Blood. 2005 Jan 1;105(1):61-6. Epub 2004 Aug 12.

PMID:
15308575
[PubMed - indexed for MEDLINE]
Free Article
16.

Biodistribution of the RD114/mammalian type D retrovirus receptor, RDR.

Green BJ, Lee CS, Rasko JE.

J Gene Med. 2004 Mar;6(3):249-59.

PMID:
15026986
[PubMed - indexed for MEDLINE]
17.

Long-term clinical and molecular follow-up of large animals receiving retrovirally transduced stem and progenitor cells: no progression to clonal hematopoiesis or leukemia.

Kiem HP, Sellers S, Thomasson B, Morris JC, Tisdale JF, Horn PA, Hematti P, Adler R, Kuramoto K, Calmels B, Bonifacino A, Hu J, von Kalle C, Schmidt M, Sorrentino B, Nienhuis A, Blau CA, Andrews RG, Donahue RE, Dunbar CE.

Mol Ther. 2004 Mar;9(3):389-95.

PMID:
15006605
[PubMed - indexed for MEDLINE]
18.

Gene therapy insertional mutagenesis insights.

Davé UP, Jenkins NA, Copeland NG.

Science. 2004 Jan 16;303(5656):333. No abstract available.

PMID:
14726584
[PubMed - indexed for MEDLINE]
Free Article
19.

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1.

Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, Lim A, Osborne CS, Pawliuk R, Morillon E, Sorensen R, Forster A, Fraser P, Cohen JI, de Saint Basile G, Alexander I, Wintergerst U, Frebourg T, Aurias A, Stoppa-Lyonnet D, Romana S, Radford-Weiss I, Gross F, Valensi F, Delabesse E, Macintyre E, Sigaux F, Soulier J, Leiva LE, Wissler M, Prinz C, Rabbitts TH, Le Deist F, Fischer A, Cavazzana-Calvo M.

Science. 2003 Oct 17;302(5644):415-9. Erratum in: Science. 2003 Oct 24;302(5645):568.

PMID:
14564000
[PubMed - indexed for MEDLINE]
Free Article
20.

Concentrated RD114-pseudotyped MFGS-gp91phox vector achieves high levels of functional correction of the chronic granulomatous disease oxidase defect in NOD/SCID/beta -microglobulin-/- repopulating mobilized human peripheral blood CD34+ cells.

Brenner S, Whiting-Theobald NL, Linton GF, Holmes KL, Anderson-Cohen M, Kelly PF, Vanin EF, Pilon AM, Bodine DM, Horwitz ME, Malech HL.

Blood. 2003 Oct 15;102(8):2789-97. Epub 2003 Jun 26.

PMID:
12829597
[PubMed - indexed for MEDLINE]
Free Article

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