240 related articles for article (PubMed ID: 19259069)
1. Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models.
Marangoni F; Bosticardo M; Charrier S; Draghici E; Locci M; Scaramuzza S; Panaroni C; Ponzoni M; Sanvito F; Doglioni C; Liabeuf M; Gjata B; Montus M; Siminovitch K; Aiuti A; Naldini L; Dupré L; Roncarolo MG; Galy A; Villa A
Mol Ther; 2009 Jun; 17(6):1073-82. PubMed ID: 19259069
[TBL] [Abstract][Full Text] [Related]
2. Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study.
Ferrua F; Cicalese MP; Galimberti S; Giannelli S; Dionisio F; Barzaghi F; Migliavacca M; Bernardo ME; Calbi V; Assanelli AA; Facchini M; Fossati C; Albertazzi E; Scaramuzza S; Brigida I; Scala S; Basso-Ricci L; Pajno R; Casiraghi M; Canarutto D; Salerio FA; Albert MH; Bartoli A; Wolf HM; Fiori R; Silvani P; Gattillo S; Villa A; Biasco L; Dott C; Culme-Seymour EJ; van Rossem K; Atkinson G; Valsecchi MG; Roncarolo MG; Ciceri F; Naldini L; Aiuti A
Lancet Haematol; 2019 May; 6(5):e239-e253. PubMed ID: 30981783
[TBL] [Abstract][Full Text] [Related]
3. Efficacy of gene therapy for Wiskott-Aldrich syndrome using a WAS promoter/cDNA-containing lentiviral vector and nonlethal irradiation.
Dupré L; Marangoni F; Scaramuzza S; Trifari S; Hernández RJ; Aiuti A; Naldini L; Roncarolo MG
Hum Gene Ther; 2006 Mar; 17(3):303-13. PubMed ID: 16544979
[TBL] [Abstract][Full Text] [Related]
4. Ubiquitous high-level gene expression in hematopoietic lineages provides effective lentiviral gene therapy of murine Wiskott-Aldrich syndrome.
Astrakhan A; Sather BD; Ryu BY; Khim S; Singh S; Humblet-Baron S; Ochs HD; Miao CH; Rawlings DJ
Blood; 2012 May; 119(19):4395-407. PubMed ID: 22431569
[TBL] [Abstract][Full Text] [Related]
5. Lentiviral-mediated gene therapy leads to improvement of B-cell functionality in a murine model of Wiskott-Aldrich syndrome.
Bosticardo M; Draghici E; Schena F; Sauer AV; Fontana E; Castiello MC; Catucci M; Locci M; Naldini L; Aiuti A; Roncarolo MG; Poliani PL; Traggiai E; Villa A
J Allergy Clin Immunol; 2011 Jun; 127(6):1376-84.e5. PubMed ID: 21531013
[TBL] [Abstract][Full Text] [Related]
6. B-cell reconstitution after lentiviral vector-mediated gene therapy in patients with Wiskott-Aldrich syndrome.
Castiello MC; Scaramuzza S; Pala F; Ferrua F; Uva P; Brigida I; Sereni L; van der Burg M; Ottaviano G; Albert MH; Grazia Roncarolo M; Naldini L; Aiuti A; Villa A; Bosticardo M
J Allergy Clin Immunol; 2015 Sep; 136(3):692-702.e2. PubMed ID: 25792466
[TBL] [Abstract][Full Text] [Related]
7. Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.
Braun CJ; Boztug K; Paruzynski A; Witzel M; Schwarzer A; Rothe M; Modlich U; Beier R; Göhring G; Steinemann D; Fronza R; Ball CR; Haemmerle R; Naundorf S; Kühlcke K; Rose M; Fraser C; Mathias L; Ferrari R; Abboud MR; Al-Herz W; Kondratenko I; Maródi L; Glimm H; Schlegelberger B; Schambach A; Albert MH; Schmidt M; von Kalle C; Klein C
Sci Transl Med; 2014 Mar; 6(227):227ra33. PubMed ID: 24622513
[TBL] [Abstract][Full Text] [Related]
8. Defects in T-cell-mediated immunity to influenza virus in murine Wiskott-Aldrich syndrome are corrected by oncoretroviral vector-mediated gene transfer into repopulating hematopoietic cells.
Strom TS; Turner SJ; Andreansky S; Liu H; Doherty PC; Srivastava DK; Cunningham JM; Nienhuis AW
Blood; 2003 Nov; 102(9):3108-16. PubMed ID: 12855574
[TBL] [Abstract][Full Text] [Related]
9. A lentiviral vector encoding the human Wiskott-Aldrich syndrome protein corrects immune and cytoskeletal defects in WASP knockout mice.
Charrier S; Stockholm D; Seye K; Opolon P; Taveau M; Gross DA; Bucher-Laurent S; Delenda C; Vainchenker W; Danos O; Galy A
Gene Ther; 2005 Apr; 12(7):597-606. PubMed ID: 15616597
[TBL] [Abstract][Full Text] [Related]
10. 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. PubMed ID: 23786330
[TBL] [Abstract][Full Text] [Related]
11. Foamy virus vector-mediated gene correction of a mouse model of Wiskott-Aldrich syndrome.
Uchiyama T; Adriani M; Jagadeesh GJ; Paine A; Candotti F
Mol Ther; 2012 Jun; 20(6):1270-9. PubMed ID: 22215016
[TBL] [Abstract][Full Text] [Related]
12. Hematopoietic-specific lentiviral vectors circumvent cellular toxicity due to ectopic expression of Wiskott-Aldrich syndrome protein.
Toscano MG; Frecha C; Benabdellah K; Cobo M; Blundell M; Thrasher AJ; García-Olivares E; Molina IJ; Martin F
Hum Gene Ther; 2008 Feb; 19(2):179-97. PubMed ID: 18240968
[TBL] [Abstract][Full Text] [Related]
13. Dendritic cell functional improvement in a preclinical model of lentiviral-mediated gene therapy for Wiskott-Aldrich syndrome.
Catucci M; Prete F; Bosticardo M; Castiello MC; Draghici E; Locci M; Roncarolo MG; Aiuti A; Benvenuti F; Villa A
Gene Ther; 2012 Dec; 19(12):1150-8. PubMed ID: 22189416
[TBL] [Abstract][Full Text] [Related]
14. Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients.
Charrier S; Dupré L; Scaramuzza S; Jeanson-Leh L; Blundell MP; Danos O; Cattaneo F; Aiuti A; Eckenberg R; Thrasher AJ; Roncarolo MG; Galy A
Gene Ther; 2007 Mar; 14(5):415-28. PubMed ID: 17051251
[TBL] [Abstract][Full Text] [Related]
15. Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.
Aiuti A; Biasco L; Scaramuzza S; Ferrua F; Cicalese MP; Baricordi C; Dionisio F; Calabria A; Giannelli S; Castiello MC; Bosticardo M; Evangelio C; Assanelli A; Casiraghi M; Di Nunzio S; Callegaro L; Benati C; Rizzardi P; Pellin D; Di Serio C; Schmidt M; Von Kalle C; Gardner J; Mehta N; Neduva V; Dow DJ; Galy A; Miniero R; Finocchi A; Metin A; Banerjee PP; Orange JS; Galimberti S; Valsecchi MG; Biffi A; Montini E; Villa A; Ciceri F; Roncarolo MG; Naldini L
Science; 2013 Aug; 341(6148):1233151. PubMed ID: 23845947
[TBL] [Abstract][Full Text] [Related]
16. Current and emerging treatment options for Wiskott-Aldrich syndrome.
Worth AJ; Thrasher AJ
Expert Rev Clin Immunol; 2015; 11(9):1015-32. PubMed ID: 26159751
[TBL] [Abstract][Full Text] [Related]
17. A peptide derived from the Wiskott-Aldrich syndrome (WAS) protein-interacting protein (WIP) restores WAS protein level and actin cytoskeleton reorganization in lymphocytes from patients with WAS mutations that disrupt WIP binding.
Massaad MJ; Ramesh N; Le Bras S; Giliani S; Notarangelo LD; Al-Herz W; Notarangelo LD; Geha RS
J Allergy Clin Immunol; 2011 Apr; 127(4):998-1005.e1-2. PubMed ID: 21376381
[TBL] [Abstract][Full Text] [Related]
18. Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome.
Hacein-Bey Abina S; Gaspar HB; Blondeau J; Caccavelli L; Charrier S; Buckland K; Picard C; Six E; Himoudi N; Gilmour K; McNicol AM; Hara H; Xu-Bayford J; Rivat C; Touzot F; Mavilio F; Lim A; Treluyer JM; Héritier S; Lefrère F; Magalon J; Pengue-Koyi I; Honnet G; Blanche S; Sherman EA; Male F; Berry C; Malani N; Bushman FD; Fischer A; Thrasher AJ; Galy A; Cavazzana M
JAMA; 2015 Apr; 313(15):1550-63. PubMed ID: 25898053
[TBL] [Abstract][Full Text] [Related]
19. Lentiviral vector-mediated gene transfer in T cells from Wiskott-Aldrich syndrome patients leads to functional correction.
Dupré L; Trifari S; Follenzi A; Marangoni F; Lain de Lera T; Bernad A; Martino S; Tsuchiya S; Bordignon C; Naldini L; Aiuti A; Roncarolo MG
Mol Ther; 2004 Nov; 10(5):903-15. PubMed ID: 15509508
[TBL] [Abstract][Full Text] [Related]
20. Gene therapy for Wiskott-Aldrich syndrome: rescue of T-cell signaling and amelioration of colitis upon transplantation of retrovirally transduced hematopoietic stem cells in mice.
Klein C; Nguyen D; Liu CH; Mizoguchi A; Bhan AK; Miki H; Takenawa T; Rosen FS; Alt FW; Mulligan RC; Snapper SB
Blood; 2003 Mar; 101(6):2159-66. PubMed ID: 12433691
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
