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7. Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial. Sessa M; Lorioli L; Fumagalli F; Acquati S; Redaelli D; Baldoli C; Canale S; Lopez ID; Morena F; Calabria A; Fiori R; Silvani P; Rancoita PM; Gabaldo M; Benedicenti F; Antonioli G; Assanelli A; Cicalese MP; Del Carro U; Sora MG; Martino S; Quattrini A; Montini E; Di Serio C; Ciceri F; Roncarolo MG; Aiuti A; Naldini L; Biffi A Lancet; 2016 Jul; 388(10043):476-87. PubMed ID: 27289174 [TBL] [Abstract][Full Text] [Related]
8. Metachromatic leukodystrophy: Disease spectrum and approaches for treatment. van Rappard DF; Boelens JJ; Wolf NI Best Pract Res Clin Endocrinol Metab; 2015 Mar; 29(2):261-73. PubMed ID: 25987178 [TBL] [Abstract][Full Text] [Related]
9. Defibrotide for Treatment of Severe Veno-Occlusive Disease in Pediatrics and Adults: An Exploratory Analysis Using Data from the Center for International Blood and Marrow Transplant Research. Strouse C; Richardson P; Prentice G; Korman S; Hume R; Nejadnik B; Horowitz MM; Saber W Biol Blood Marrow Transplant; 2016 Jul; 22(7):1306-1312. PubMed ID: 27108694 [TBL] [Abstract][Full Text] [Related]
10. Medicine. Gene therapy that works. Verma IM Science; 2013 Aug; 341(6148):853-5. PubMed ID: 23970689 [No Abstract] [Full Text] [Related]
11. Juvenile metachromatic leukodystrophy: understanding the disease and implications for nursing care. Barrell C J Pediatr Oncol Nurs; 2007; 24(2):64-9. PubMed ID: 17332420 [TBL] [Abstract][Full Text] [Related]
12. Hepatic veno-occlusive disease in pediatric stem cell transplantation: impact of pre-emptive antithrombin III replacement and combined antithrombin III/defibrotide therapy. Haussmann U; Fischer J; Eber S; Scherer F; Seger R; Gungor T Haematologica; 2006 Jun; 91(6):795-800. PubMed ID: 16769582 [TBL] [Abstract][Full Text] [Related]
13. Broadening the indications for hematopoietic stem cell genetic therapies. Williams DA Cell Stem Cell; 2013 Sep; 13(3):263-4. PubMed ID: 24012366 [TBL] [Abstract][Full Text] [Related]
14. Gene therapy: primed for take-off. Leboulch P Nature; 2013 Aug; 500(7462):280-2. PubMed ID: 23955226 [No Abstract] [Full Text] [Related]
15. Allogeneic hematopoietic cell transplantation in systemic mastocytosis: is there a high risk for veno-occlusive disease? Ustun C; Smith A; Cayci Z; Courville EL; Corbacioglu S; Akin C; Horny HP; Valent P; Devine S; Weisdorf DJ Eur J Haematol; 2016 Jun; 96(6):655-7. PubMed ID: 26679533 [No Abstract] [Full Text] [Related]
16. Reply to: Reduced intensity conditioning haematopoietic stem cell transplantation with mesenchymal stromal cells infusion for the treatment of metachromatic leukodystrophy: a case report. Haematologica 2008; 93:e11-13. Charnas L; Eichler F; Kohlschuetter A; Tolar J; Orchard PJ Haematologica; 2008 May; 93(5):e47; author reply e48. PubMed ID: 18450730 [No Abstract] [Full Text] [Related]