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Items: 1 to 20 of 131

1.

Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis.

Chiriaco M, Farinelli G, Capo V, Zonari E, Scaramuzza S, Di Matteo G, Sergi LS, Migliavacca M, Hernandez RJ, Bombelli F, Giorda E, Kajaste-Rudnitski A, Trono D, Grez M, Rossi P, Finocchi A, Naldini L, Gentner B, Aiuti A.

Mol Ther. 2014 Aug;22(8):1472-83. doi: 10.1038/mt.2014.87. Epub 2014 May 29.

2.

Human miR223 promoter as a novel myelo-specific promoter for chronic granulomatous disease gene therapy.

Brendel C, Hänseler W, Wohlgensinger V, Bianchi M, Tokmak S, Chen-Wichmann L, Kuzmenko E, Cesarovic N, Nicholls F, Reichenbach J, Seger R, Grez M, Siler U.

Hum Gene Ther Methods. 2013 Jun;24(3):151-9. doi: 10.1089/hgtb.2012.157. Epub 2013 May 2.

3.

Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells.

Santilli G, Almarza E, Brendel C, Choi U, Beilin C, Blundell MP, Haria S, Parsley KL, Kinnon C, Malech HL, Bueren JA, Grez M, Thrasher AJ.

Mol Ther. 2011 Jan;19(1):122-32. doi: 10.1038/mt.2010.226. Epub 2010 Oct 26.

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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.

6.

Correction of respiratory burst activity in X-linked chronic granulomatous cells to therapeutically relevant levels after gene transfer into bone marrow CD34+ cells.

Becker S, Wasser S, Hauses M, Hossle JP, Ott MG, Dinauer MC, Ganser A, Hoelzer D, Seger R, Grez M.

Hum Gene Ther. 1998 Jul 20;9(11):1561-70.

PMID:
9694155
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.

8.

Drug-selected co-expression of P-glycoprotein and gp91 in vivo from an MDR1-bicistronic retrovirus vector Ha-MDR-IRES-gp91.

Sugimoto Y, Tsukahara S, Sato S, Suzuki M, Nunoi H, Malech HL, Gottesman MM, Tsuruo T.

J Gene Med. 2003 May;5(5):366-76.

PMID:
12731085
9.

Lineage- and stage-restricted lentiviral vectors for the gene therapy of chronic granulomatous disease.

Barde I, Laurenti E, Verp S, Wiznerowicz M, Offner S, Viornery A, Galy A, Trumpp A, Trono D.

Gene Ther. 2011 Nov;18(11):1087-97. doi: 10.1038/gt.2011.65. Epub 2011 May 5.

PMID:
21544095
10.

From bench to bedside: preclinical evaluation of a self-inactivating gammaretroviral vector for the gene therapy of X-linked chronic granulomatous disease.

Stein S, Scholz S, Schwäble J, Sadat MA, Modlich U, Schultze-Strasser S, Diaz M, Chen-Wichmann L, Müller-Kuller U, Brendel C, Fronza R, Kaufmann KB, Naundorf S, Pech NK, Travers JB, Matute JD, Presson RG Jr, Sandusky GE, Kunkel H, Rudolf E, Dillmann A, von Kalle C, Kühlcke K, Baum C, Schambach A, Dinauer MC, Schmidt M, Grez M.

Hum Gene Ther Clin Dev. 2013 Jun;24(2):86-98. doi: 10.1089/humc.2013.019.

PMID:
23845071
11.

Transgene optimization significantly improves SIN vector titers, gp91phox expression and reconstitution of superoxide production in X-CGD cells.

Moreno-Carranza B, Gentsch M, Stein S, Schambach A, Santilli G, Rudolf E, Ryser MF, Haria S, Thrasher AJ, Baum C, Brenner S, Grez M.

Gene Ther. 2009 Jan;16(1):111-8. doi: 10.1038/gt.2008.143. Epub 2008 Sep 11.

PMID:
18784749
12.

Chronic granulomatous disease: Clinical, molecular, and therapeutic aspects.

Chiriaco M, Salfa I, Di Matteo G, Rossi P, Finocchi A.

Pediatr Allergy Immunol. 2016 May;27(3):242-53. doi: 10.1111/pai.12527. Epub 2016 Jan 21. Review.

PMID:
26680691
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18.

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
19.

Gene therapy of chronic granulomatous disease.

Grez M, Becker S, Saulnier S, Knöss H, Ott MG, Maurer A, Dinauer MC, Hoelzer D, Seger R, Hossle JP.

Bone Marrow Transplant. 2000 May;25 Suppl 2:S99-104.

PMID:
10933200
20.

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.

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