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Items: 1 to 20 of 48

1.

Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

Aiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani B, Callegaro L, Scaramuzza S, Andolfi G, Mirolo M, Brigida I, Tabucchi A, Carlucci F, Eibl M, Aker M, Slavin S, Al-Mousa H, Al Ghonaium A, Ferster A, Duppenthaler A, Notarangelo L, Wintergerst U, Buckley RH, Bregni M, Marktel S, Valsecchi MG, Rossi P, Ciceri F, Miniero R, Bordignon C, Roncarolo MG.

N Engl J Med. 2009 Jan 29;360(5):447-58. doi: 10.1056/NEJMoa0805817.

2.

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients.

Howe SJ, Mansour MR, Schwarzwaelder K, Bartholomae C, Hubank M, Kempski H, Brugman MH, Pike-Overzet K, Chatters SJ, de Ridder D, Gilmour KC, Adams S, Thornhill SI, Parsley KL, Staal FJ, Gale RE, Linch DC, Bayford J, Brown L, Quaye M, Kinnon C, Ancliff P, Webb DK, Schmidt M, von Kalle C, Gaspar HB, Thrasher AJ.

J Clin Invest. 2008 Sep;118(9):3143-50. doi: 10.1172/JCI35798.

3.

Cognitive and behavioral abnormalities in children after hematopoietic stem cell transplantation for severe congenital immunodeficiencies.

Titman P, Pink E, Skucek E, O'Hanlon K, Cole TJ, Gaspar J, Xu-Bayford J, Jones A, Thrasher AJ, Davies EG, Veys PA, Gaspar HB.

Blood. 2008 Nov 1;112(9):3907-13. doi: 10.1182/blood-2008-04-151332. Epub 2008 Jul 21.

4.

Neonatal bone marrow transplantation of ADA-deficient SCID mice results in immunologic reconstitution despite low levels of engraftment and an absence of selective donor T lymphoid expansion.

Carbonaro DA, Jin X, Cotoi D, Mi T, Yu XJ, Skelton DC, Dorey F, Kellems RE, Blackburn MR, Kohn DB.

Blood. 2008 Jun 15;111(12):5745-54. doi: 10.1182/blood-2007-08-103663. Epub 2008 Mar 20.

5.

Altered intracellular and extracellular signaling leads to impaired T-cell functions in ADA-SCID patients.

Cassani B, Mirolo M, Cattaneo F, Benninghoff U, Hershfield M, Carlucci F, Tabucchi A, Bordignon C, Roncarolo MG, Aiuti A.

Blood. 2008 Apr 15;111(8):4209-19. doi: 10.1182/blood-2007-05-092429. Epub 2008 Jan 24. Erratum in: Blood. 2014 Jun 5;123(23):3682.

6.

Gene therapy for childhood immunological diseases.

Kohn DB.

Bone Marrow Transplant. 2008 Jan;41(2):199-205. Epub 2007 Nov 12. Review.

PMID:
17994122
7.

Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy.

Aiuti A, Cassani B, Andolfi G, Mirolo M, Biasco L, Recchia A, Urbinati F, Valacca C, Scaramuzza S, Aker M, Slavin S, Cazzola M, Sartori D, Ambrosi A, Di Serio C, Roncarolo MG, Mavilio F, Bordignon C.

J Clin Invest. 2007 Aug;117(8):2233-40.

8.

Burkitt's lymphoma in a patient with adenosine deaminase deficiency-severe combined immunodeficiency treated with polyethylene glycol-adenosine deaminase.

Husain M, Grunebaum E, Naqvi A, Atkinson A, Ngan BY, Aiuti A, Roifman CM.

J Pediatr. 2007 Jul;151(1):93-5.

PMID:
17586199
9.

Management options for adenosine deaminase deficiency; proceedings of the EBMT satellite workshop (Hamburg, March 2006).

Booth C, Hershfield M, Notarangelo L, Buckley R, Hoenig M, Mahlaoui N, Cavazzana-Calvo M, Aiuti A, Gaspar HB.

Clin Immunol. 2007 May;123(2):139-47. Epub 2007 Feb 14. Review.

PMID:
17300989
10.

Patients with adenosine deaminase deficiency surviving after hematopoietic stem cell transplantation are at high risk of CNS complications.

Hönig M, Albert MH, Schulz A, Sparber-Sauer M, Schütz C, Belohradsky B, Güngör T, Rojewski MT, Bode H, Pannicke U, Lippold D, Schwarz K, Debatin KM, Hershfield MS, Friedrich W.

Blood. 2007 Apr 15;109(8):3595-602. Epub 2006 Dec 21.

11.

Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report.

Engel BC, Podsakoff GM, Ireland JL, Smogorzewska EM, Carbonaro DA, Wilson K, Shah A, Kapoor N, Sweeney M, Borchert M, Crooks GM, Weinberg KI, Parkman R, Rosenblatt HM, Wu SQ, Hershfield MS, Candotti F, Kohn DB.

Blood. 2007 Jan 15;109(2):503-6. Epub 2006 Sep 14.

12.

Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning.

Gaspar HB, Bjorkegren E, Parsley K, Gilmour KC, King D, Sinclair J, Zhang F, Giannakopoulos A, Adams S, Fairbanks LD, Gaspar J, Henderson L, Xu-Bayford JH, Davies EG, Veys PA, Kinnon C, Thrasher AJ.

Mol Ther. 2006 Oct;14(4):505-13. Epub 2006 Aug 14.

13.

Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)-deficient mice and corrects their immune and metabolic defects.

Mortellaro A, Hernandez RJ, Guerrini MM, Carlucci F, Tabucchi A, Ponzoni M, Sanvito F, Doglioni C, Di Serio C, Biasco L, Follenzi A, Naldini L, Bordignon C, Roncarolo MG, Aiuti A.

Blood. 2006 Nov 1;108(9):2979-88. Epub 2006 Jul 11.

14.

Bone marrow transplantation for severe combined immune deficiency.

Grunebaum E, Mazzolari E, Porta F, Dallera D, Atkinson A, Reid B, Notarangelo LD, Roifman CM.

JAMA. 2006 Feb 1;295(5):508-18.

PMID:
16449616
15.

Reduced thymic output, increased spontaneous apoptosis and oligoclonal B cells in polyethylene glycol-adenosine deaminase-treated patients.

Malacarne F, Benicchi T, Notarangelo LD, Mori L, Parolini S, Caimi L, Hershfield M, Notarangelo LD, Imberti L.

Eur J Immunol. 2005 Nov;35(11):3376-86.

16.

Cerebral lymphoma in an adenosine deaminase-deficient patient with severe combined immunodeficiency receiving polyethylene glycol-conjugated adenosine deaminase.

Kaufman DA, Hershfield MS, Bocchini JA, Moissidis IJ, Jeroudi M, Bahna SL.

Pediatrics. 2005 Dec;116(6):e876-9. Epub 2005 Nov 1.

PMID:
16263974
17.

Long-term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID).

Chan B, Wara D, Bastian J, Hershfield MS, Bohnsack J, Azen CG, Parkman R, Weinberg K, Kohn DB.

Clin Immunol. 2005 Nov;117(2):133-43. Epub 2005 Aug 22.

PMID:
16112907
18.

polyethylene glycol-conjugated adenosine deaminase (ADA) therapy provides temporary immune reconstitution to a child with delayed-onset ADA deficiency.

Lainka E, Hershfield MS, Santisteban I, Bali P, Seibt A, Neubert J, Friedrich W, Niehues T.

Clin Diagn Lab Immunol. 2005 Jul;12(7):861-6.

19.

Severe combined immunodeficiency. A model disease for molecular immunology and therapy.

Fischer A, Le Deist F, Hacein-Bey-Abina S, André-Schmutz I, Basile Gde S, de Villartay JP, Cavazzana-Calvo M.

Immunol Rev. 2005 Feb;203:98-109. Review.

PMID:
15661024
20.

Clonal evidence for the transduction of CD34+ cells with lymphomyeloid differentiation potential and self-renewal capacity in the SCID-X1 gene therapy trial.

Schmidt M, Hacein-Bey-Abina S, Wissler M, Carlier F, Lim A, Prinz C, Glimm H, Andre-Schmutz I, Hue C, Garrigue A, Le Deist F, Lagresle C, Fischer A, Cavazzana-Calvo M, von Kalle C.

Blood. 2005 Apr 1;105(7):2699-706. Epub 2004 Dec 7.

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