235 related articles for article (PubMed ID: 28716862)
1. Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult.
Morris EC; Fox T; Chakraverty R; Tendeiro R; Snell K; Rivat C; Grace S; Gilmour K; Workman S; Buckland K; Butler K; Chee R; Salama AD; Ibrahim H; Hara H; Duret C; Mavilio F; Male F; Bushman FD; Galy A; Burns SO; Gaspar HB; Thrasher AJ
Blood; 2017 Sep; 130(11):1327-1335. PubMed ID: 28716862
[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. 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. 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]
5. 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]
6. 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]
7. 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]
8. Ten years of gene therapy for primary immune deficiencies.
Aiuti A; Roncarolo MG
Hematology Am Soc Hematol Educ Program; 2009; ():682-9. PubMed ID: 20008254
[TBL] [Abstract][Full Text] [Related]
9. 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]
10. Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome.
Labrosse R; Chu JI; Armant MA; Everett JK; Pellin D; Kareddy N; Frelinger AL; Henderson LA; O'Connell AE; Biswas A; Coenen-van der Spek J; Miggelbrink A; Fiorini C; Adhikari H; Berry CC; Cantu VA; Fong J; Jaroslavsky J; Karadeniz DF; Li QZ; Reddy S; Roche AM; Zhu C; Whangbo JS; Dansereau C; Mackinnon B; Morris E; Koo SM; London WB; Baris S; Ozen A; Karakoc-Aydiner E; Despotovic JM; Forbes Satter LR; Saitoh A; Aizawa Y; King A; Nguyen MAT; Vu VDU; Snapper SB; Galy A; Notarangelo LD; Bushman FD; Williams DA; Pai SY
Blood; 2023 Oct; 142(15):1281-1296. PubMed ID: 37478401
[TBL] [Abstract][Full Text] [Related]
11. 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]
12. 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]
13. 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]
14. 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]
15. Stem-cell gene therapy for the Wiskott-Aldrich syndrome.
Boztug K; Schmidt M; Schwarzer A; Banerjee PP; Díez IA; Dewey RA; Böhm M; Nowrouzi A; Ball CR; Glimm H; Naundorf S; Kühlcke K; Blasczyk R; Kondratenko I; Maródi L; Orange JS; von Kalle C; Klein C
N Engl J Med; 2010 Nov; 363(20):1918-27. PubMed ID: 21067383
[TBL] [Abstract][Full Text] [Related]
16. Gene therapy for Wiskott-Aldrich Syndrome.
Bosticardo M; Ferrua F; Cavazzana M; Aiuti A
Curr Gene Ther; 2014; 14(6):413-21. PubMed ID: 25245089
[TBL] [Abstract][Full Text] [Related]
17. 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]
18. Development of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome.
Boztug K; Dewey RA; Klein C
Curr Opin Mol Ther; 2006 Oct; 8(5):390-5. PubMed ID: 17078381
[TBL] [Abstract][Full Text] [Related]
19. 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]
20. 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]
[Next] [New Search]
