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Items: 1 to 20 of 149

1.

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 11;363(20):1918-27. doi: 10.1056/NEJMoa1003548.

2.

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 21;313(15):1550-63. doi: 10.1001/jama.2015.3253.

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. doi: 10.1016/j.jaci.2015.01.035. Epub 2015 Mar 16.

4.

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 12;6(227):227ra33. doi: 10.1126/scitranslmed.3007280.

5.

Gene therapy for Wiskott-Aldrich Syndrome.

Bosticardo M, Ferrua F, Cavazzana M, Aiuti A.

Curr Gene Ther. 2014;14(6):413-21. Review.

PMID:
25245089
6.

Current and emerging treatment options for Wiskott-Aldrich syndrome.

Worth AJ, Thrasher AJ.

Expert Rev Clin Immunol. 2015;11(9):1015-32. doi: 10.1586/1744666X.2015.1062366. Epub 2015 Jul 9. Review.

PMID:
26159751
7.

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.

PMID:
16544979
8.

Wiskott-Aldrich syndrome.

Notarangelo LD, Miao CH, Ochs HD.

Curr Opin Hematol. 2008 Jan;15(1):30-6. Review.

PMID:
18043243
9.

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. doi: 10.1038/mt.2011.282. Epub 2012 Jan 3.

10.

[Wiskott-Aldrich syndrome].

Gulácsy V, Maródi L.

Orv Hetil. 2008 Jul 20;149(29):1367-71. doi: 10.1556/OH.2008.28377. Review. Hungarian.

PMID:
18617469
11.

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 1;102(9):3108-16. Epub 2003 Jul 10.

12.

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
13.

New insights into the biology of Wiskott-Aldrich syndrome (WAS).

Thrasher AJ.

Hematology Am Soc Hematol Educ Program. 2009:132-8. doi: 10.1182/asheducation-2009.1.132. Review.

PMID:
20008191
14.

The genotype of the original Wiskott phenotype.

Binder V, Albert MH, Kabus M, Bertone M, Meindl A, Belohradsky BH.

N Engl J Med. 2006 Oct 26;355(17):1790-3.

15.

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. Review.

PMID:
17078381
16.

Recent advances in understanding the pathophysiology of Wiskott-Aldrich syndrome.

Bosticardo M, Marangoni F, Aiuti A, Villa A, Grazia Roncarolo M.

Blood. 2009 Jun 18;113(25):6288-95. doi: 10.1182/blood-2008-12-115253. Epub 2009 Apr 7. Review.

17.

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. doi: 10.1016/j.jaci.2011.03.030. Epub 2011 Apr 29.

PMID:
21531013
18.

An emerging era of clinical benefit from gene therapy.

Malech HL, Ochs HD.

JAMA. 2015 Apr 21;313(15):1522-3. doi: 10.1001/jama.2015.2055. No abstract available.

PMID:
25898049
19.
20.

Stable mixed chimerism after hematopoietic stem cell transplantation in Wiskott-Aldrich syndrome.

Doğu F, Kurtuluş-Ulküer M, Bilge Y, Bozdoğan G, Ulküer U, Malhatun E, Ikincioğullari A, Babacan E.

Pediatr Transplant. 2006 May;10(3):395-9.

PMID:
16677369

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