BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

184 related articles for article (PubMed ID: 32623069)

  • 1. Gene therapy for Wiskott-Aldrich syndrome: History, new vectors, future directions.
    Ferrua F; Marangoni F; Aiuti A; Roncarolo MG
    J Allergy Clin Immunol; 2020 Aug; 146(2):262-265. PubMed ID: 32623069
    [No 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. 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]  

  • 4. 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]  

  • 5. 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]  

  • 6. 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]  

  • 7. Lentiviral vectors for the treatment of Wiskott-Aldrich syndrome.
    Nienhuis AW
    Gene Ther; 2005 Apr; 12(7):555-6. PubMed ID: 18360950
    [No Abstract]   [Full Text] [Related]  

  • 8. 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]  

  • 9. 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]  

  • 10. A possible turning point in the hematopoietic stem cell gene therapy for primary immunodeficiency diseases? Lentiviral vectors could take the place of retroviral vectors.
    Ariga T
    Expert Rev Clin Immunol; 2013 Nov; 9(11):1015-8. PubMed ID: 24168409
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Gene therapy for the Wiskott-Aldrich syndrome.
    Galy A; Thrasher AJ
    Curr Opin Allergy Clin Immunol; 2011 Dec; 11(6):545-50. PubMed ID: 21971332
    [TBL] [Abstract][Full Text] [Related]  

  • 12. 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]  

  • 13. Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome.
    Magnani A; Semeraro M; Adam F; Booth C; Dupré L; Morris EC; Gabrion A; Roudaut C; Borgel D; Toubert A; Clave E; Abdo C; Gorochov G; Petermann R; Guiot M; Miyara M; Moshous D; Magrin E; Denis A; Suarez F; Lagresle C; Roche AM; Everett J; Trinquand A; Guisset M; Bayford JX; Hacein-Bey-Abina S; Kauskot A; Elfeky R; Rivat C; Abbas S; Gaspar HB; Macintyre E; Picard C; Bushman FD; Galy A; Fischer A; Six E; Thrasher AJ; Cavazzana M
    Nat Med; 2022 Jan; 28(1):71-80. PubMed ID: 35075289
    [TBL] [Abstract][Full Text] [Related]  

  • 14. 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]  

  • 15. Trial watch: Clinical trial boost for lentiviral gene therapy.
    Flight MH
    Nat Rev Drug Discov; 2013 Sep; 12(9):654. PubMed ID: 23989781
    [No Abstract]   [Full Text] [Related]  

  • 16. 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]  

  • 17. Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome.
    Rai R; Romito M; Rivers E; Turchiano G; Blattner G; Vetharoy W; Ladon D; Andrieux G; Zhang F; Zinicola M; Leon-Rico D; Santilli G; Thrasher AJ; Cavazza A
    Nat Commun; 2020 Aug; 11(1):4034. PubMed ID: 32788576
    [TBL] [Abstract][Full Text] [Related]  

  • 18. 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]  

  • 19. 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]  

  • 20. Gene Therapies for Primary Immune Deficiencies.
    Kohn LA; Kohn DB
    Front Immunol; 2021; 12():648951. PubMed ID: 33717203
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 10.