236 related articles for article (PubMed ID: 38688902)
1. A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID.
Cesana D; Cicalese MP; Calabria A; Merli P; Caruso R; Volpin M; Rudilosso L; Migliavacca M; Barzaghi F; Fossati C; Gazzo F; Pizzi S; Ciolfi A; Bruselles A; Tucci F; Spinozzi G; Pais G; Benedicenti F; Barcella M; Merelli I; Gallina P; Giannelli S; Dionisio F; Scala S; Casiraghi M; Strocchio L; Vinti L; Pacillo L; Draghi E; Cesana M; Riccardo S; Colantuono C; Six E; Cavazzana M; Carlucci F; Schmidt M; Cancrini C; Ciceri F; Vago L; Cacchiarelli D; Gentner B; Naldini L; Tartaglia M; Montini E; Locatelli F; Aiuti A
Nat Commun; 2024 Apr; 15(1):3662. PubMed ID: 38688902
[TBL] [Abstract][Full Text] [Related]
2. Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety.
Cicalese MP; Ferrua F; Castagnaro L; Rolfe K; De Boever E; Reinhardt RR; Appleby J; Roncarolo MG; Aiuti A
Mol Ther; 2018 Mar; 26(3):917-931. PubMed ID: 29433935
[TBL] [Abstract][Full Text] [Related]
3. Outcomes in two Japanese adenosine deaminase-deficiency patients treated by stem cell gene therapy with no cytoreductive conditioning.
Otsu M; Yamada M; Nakajima S; Kida M; Maeyama Y; Hatano N; Toita N; Takezaki S; Okura Y; Kobayashi R; Matsumoto Y; Tatsuzawa O; Tsuchida F; Kato S; Kitagawa M; Mineno J; Hershfield MS; Bali P; Candotti F; Onodera M; Kawamura N; Sakiyama Y; Ariga T
J Clin Immunol; 2015 May; 35(4):384-98. PubMed ID: 25875699
[TBL] [Abstract][Full Text] [Related]
4. Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiency.
Shaw KL; Garabedian E; Mishra S; Barman P; Davila A; Carbonaro D; Shupien S; Silvin C; Geiger S; Nowicki B; Smogorzewska EM; Brown B; Wang X; de Oliveira S; Choi Y; Ikeda A; Terrazas D; Fu PY; Yu A; Fernandez BC; Cooper AR; Engel B; Podsakoff G; Balamurugan A; Anderson S; Muul L; Jagadeesh GJ; Kapoor N; Tse J; Moore TB; Purdy K; Rishi R; Mohan K; Skoda-Smith S; Buchbinder D; Abraham RS; Scharenberg A; Yang OO; Cornetta K; Gjertson D; Hershfield M; Sokolic R; Candotti F; Kohn DB
J Clin Invest; 2017 May; 127(5):1689-1699. PubMed ID: 28346229
[TBL] [Abstract][Full Text] [Related]
5. Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients.
Cooper AR; Lill GR; Shaw K; Carbonaro-Sarracino DA; Davila A; Sokolic R; Candotti F; Pellegrini M; Kohn DB
Blood; 2017 May; 129(19):2624-2635. PubMed ID: 28351939
[TBL] [Abstract][Full Text] [Related]
6. Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency.
Cicalese MP; Ferrua F; Castagnaro L; Pajno R; Barzaghi F; Giannelli S; Dionisio F; Brigida I; Bonopane M; Casiraghi M; Tabucchi A; Carlucci F; Grunebaum E; Adeli M; Bredius RG; Puck JM; Stepensky P; Tezcan I; Rolfe K; De Boever E; Reinhardt RR; Appleby J; Ciceri F; Roncarolo MG; Aiuti A
Blood; 2016 Jul; 128(1):45-54. PubMed ID: 27129325
[TBL] [Abstract][Full Text] [Related]
7. Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency.
Carbonaro DA; Zhang L; Jin X; Montiel-Equihua C; Geiger S; Carmo M; Cooper A; Fairbanks L; Kaufman ML; Sebire NJ; Hollis RP; Blundell MP; Senadheera S; Fu PY; Sahaghian A; Chan RY; Wang X; Cornetta K; Thrasher AJ; Kohn DB; Gaspar HB
Mol Ther; 2014 Mar; 22(3):607-622. PubMed ID: 24256635
[TBL] [Abstract][Full Text] [Related]
8. Recombinant adeno-associated virus-mediated gene transfer for the potential therapy of adenosine deaminase-deficient severe combined immune deficiency.
Silver JN; Elder M; Conlon T; Cruz P; Wright AJ; Srivastava A; Flotte TR
Hum Gene Ther; 2011 Aug; 22(8):935-49. PubMed ID: 21142972
[TBL] [Abstract][Full Text] [Related]
9. Twenty-Five Years of Gene Therapy for ADA-SCID: From Bubble Babies to an Approved Drug.
Ferrua F; Aiuti A
Hum Gene Ther; 2017 Nov; 28(11):972-981. PubMed ID: 28847159
[TBL] [Abstract][Full Text] [Related]
10. Good Laboratory Practice Preclinical Safety Studies for GSK2696273 (MLV Vector-Based Ex Vivo Gene Therapy for Adenosine Deaminase Deficiency Severe Combined Immunodeficiency) in NSG Mice.
Carriglio N; Klapwijk J; Hernandez RJ; Vezzoli M; Chanut F; Lowe R; Draghici E; Nord M; Albertini P; Cristofori P; Richards J; Staton H; Appleby J; Aiuti A; Sauer AV
Hum Gene Ther Clin Dev; 2017 Mar; 28(1):17-27. PubMed ID: 28319446
[TBL] [Abstract][Full Text] [Related]
11. Update on gene therapy for adenosine deaminase-deficient severe combined immunodeficiency.
Ferrua F; Brigida I; Aiuti A
Curr Opin Allergy Clin Immunol; 2010 Dec; 10(6):551-6. PubMed ID: 20966749
[TBL] [Abstract][Full Text] [Related]
12. Promising Results with Ex Vivo Lentiviral HSPC Gene Therapy in ADA-SCID.
Am J Med Genet A; 2021 Aug; 185(8):2291-2292. PubMed ID: 34240545
[No Abstract] [Full Text] [Related]
13. Long-Term Outcome of Adenosine Deaminase-Deficient Patients-a Single-Center Experience.
Scott O; Kim VH; Reid B; Pham-Huy A; Atkinson AR; Aiuti A; Grunebaum E
J Clin Immunol; 2017 Aug; 37(6):582-591. PubMed ID: 28748310
[TBL] [Abstract][Full Text] [Related]
14. Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency.
Migliavacca M; Barzaghi F; Fossati C; Rancoita PMV; Gabaldo M; Dionisio F; Giannelli S; Salerio FA; Ferrua F; Tucci F; Calbi V; Gallo V; Recupero S; Consiglieri G; Pajno R; Sambuco M; Priolo A; Ferri C; Garella V; Monti I; Silvani P; Darin S; Casiraghi M; Corti A; Zancan S; Levi M; Cesana D; Carlucci F; Pituch-Noworolska A; AbdElaziz D; Baumann U; Finocchi A; Cancrini C; Ladogana S; Meinhardt A; Meyts I; Montin D; Notarangelo LD; Porta F; Pasquet M; Speckmann C; Stepensky P; Tommasini A; Rabusin M; Karakas Z; Galicchio M; Leonardi L; Duse M; Guner SN; Di Serio C; Ciceri F; Bernardo ME; Aiuti A; Cicalese MP
Nat Med; 2024 Feb; 30(2):488-497. PubMed ID: 38355973
[TBL] [Abstract][Full Text] [Related]
15. Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction.
Gaspar HB; Cooray S; Gilmour KC; Parsley KL; Zhang F; Adams S; Bjorkegren E; Bayford J; Brown L; Davies EG; Veys P; Fairbanks L; Bordon V; Petropoulou T; Kinnon C; Thrasher AJ
Sci Transl Med; 2011 Aug; 3(97):97ra80. PubMed ID: 21865538
[TBL] [Abstract][Full Text] [Related]
16. Gene therapy/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction.
Carbonaro DA; Jin X; Wang X; Yu XJ; Rozengurt N; Kaufman ML; Wang X; Gjertson D; Zhou Y; Blackburn MR; Kohn DB
Blood; 2012 Nov; 120(18):3677-87. PubMed ID: 22833548
[TBL] [Abstract][Full Text] [Related]
17. ADA-SCID Gene Therapy Endorsed By European Medicines Agency For Marketing Authorization.
Ylä-Herttuala S
Mol Ther; 2016 Jun; 24(6):1013-1014. PubMed ID: 27324442
[No Abstract] [Full Text] [Related]
18. Gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency.
Montiel-Equihua CA; Thrasher AJ; Gaspar HB
Curr Gene Ther; 2012 Feb; 12(1):57-65. PubMed ID: 22348551
[TBL] [Abstract][Full Text] [Related]
19. Investor Outlook: Rising from the Ashes; GSK's European Approval of Strimvelis for ADA-SCID.
Schimmer J; Breazzano S
Hum Gene Ther Clin Dev; 2016 Jun; 27(2):57-61. PubMed ID: 27267267
[TBL] [Abstract][Full Text] [Related]
20. Gene therapy for severe combined immunodeficiency caused by adenosine deaminase deficiency: improved retroviral vectors for clinical trials.
Onodera M; Nelson DM; Sakiyama Y; Candotti F; Blaese RM
Acta Haematol; 1999; 101(2):89-96. PubMed ID: 10202239
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
