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Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.
Gentner B, Tucci F, Galimberti S, Fumagalli F, De Pellegrin M, Silvani P, Camesasca C, Pontesilli S, Darin S, Ciotti F, Sarzana M, Consiglieri G, Filisetti C, Forni G, Passerini L, Tomasoni D, Cesana D, Calabria A, Spinozzi G, Cicalese MP, Calbi V, Migliavacca M, Barzaghi F, Ferrua F, Gallo V, Miglietta S, Zonari E, Cheruku PS, Forni C, Facchini M, Corti A, Gabaldo M, Zancan S, Gasperini S, Rovelli A, Boelens JJ, Jones SA, Wynn R, Baldoli C, Montini E, Gregori S, Ciceri F, Valsecchi MG, la Marca G, Parini R, Naldini L, Aiuti A, Bernardo ME; MPSI Study Group. Gentner B, et al. Among authors: wynn r. N Engl J Med. 2021 Nov 18;385(21):1929-1940. doi: 10.1056/NEJMoa2106596. N Engl J Med. 2021. PMID: 34788506 Clinical Trial.
Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome.
Grewal SS, Wynn R, Abdenur JE, Burton BK, Gharib M, Haase C, Hayashi RJ, Shenoy S, Sillence D, Tiller GE, Dudek ME, van Royen-Kerkhof A, Wraith JE, Woodard P, Young GA, Wulffraat N, Whitley CB, Peters C. Grewal SS, et al. Among authors: wynn r. Genet Med. 2005 Feb;7(2):143-6. doi: 10.1097/01.gim.0000154299.22120.6a. Genet Med. 2005. PMID: 15714083 Free article. Clinical Trial.
Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure.
Boelens JJ, Wynn RF, O'Meara A, Veys P, Bertrand Y, Souillet G, Wraith JE, Fischer A, Cavazzana-Calvo M, Sykora KW, Sedlacek P, Rovelli A, Uiterwaal CS, Wulffraat N. Boelens JJ, et al. Among authors: wynn rf. Bone Marrow Transplant. 2007 Aug;40(3):225-33. doi: 10.1038/sj.bmt.1705718. Epub 2007 May 28. Bone Marrow Transplant. 2007. PMID: 17529997 Free PMC article.
Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with hurler syndrome.
Boelens JJ, Rocha V, Aldenhoven M, Wynn R, O'Meara A, Michel G, Ionescu I, Parikh S, Prasad VK, Szabolcs P, Escolar M, Gluckman E, Cavazzana-Calvo M, Kurtzberg J; EUROCORD, Inborn error Working Party of EBMT and Duke University. Boelens JJ, et al. Among authors: wynn r. Biol Blood Marrow Transplant. 2009 May;15(5):618-25. doi: 10.1016/j.bbmt.2009.01.020. Biol Blood Marrow Transplant. 2009. PMID: 19361754 Free article.
875 results