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Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.
Kohn DB, Booth C, Shaw KL, Xu-Bayford J, Garabedian E, Trevisan V, Carbonaro-Sarracino DA, Soni K, Terrazas D, Snell K, Ikeda A, Leon-Rico D, Moore TB, Buckland KF, Shah AJ, Gilmour KC, De Oliveira S, Rivat C, Crooks GM, Izotova N, Tse J, Adams S, Shupien S, Ricketts H, Davila A, Uzowuru C, Icreverzi A, Barman P, Campo Fernandez B, Hollis RP, Coronel M, Yu A, Chun KM, Casas CE, Zhang R, Arduini S, Lynn F, Kudari M, Spezzi A, Zahn M, Heimke R, Labik I, Parrott R, Buckley RH, Reeves L, Cornetta K, Sokolic R, Hershfield M, Schmidt M, Candotti F, Malech HL, Thrasher AJ, Gaspar HB. Kohn DB, et al. Among authors: kudari m. N Engl J Med. 2021 May 27;384(21):2002-2013. doi: 10.1056/NEJMoa2027675. Epub 2021 May 11. N Engl J Med. 2021. PMID: 33974366 Free PMC article. Clinical Trial.
FDXR is a biomarker of radiation exposure in vivo.
O'Brien G, Cruz-Garcia L, Majewski M, Grepl J, Abend M, Port M, Tichý A, Sirak I, Malkova A, Donovan E, Gothard L, Boyle S, Somaiah N, Ainsbury E, Ponge L, Slosarek K, Miszczyk L, Widlak P, Green E, Patel N, Kudari M, Gleeson F, Vinnikov V, Starenkiy V, Artiukh S, Vasyliev L, Zaman A, Badie C. O'Brien G, et al. Among authors: kudari m. Sci Rep. 2018 Jan 12;8(1):684. doi: 10.1038/s41598-017-19043-w. Sci Rep. 2018. PMID: 29330481 Free PMC article.
Gene therapy in rare diseases: the benefits and challenges of developing a patient-centric registry for Strimvelis in ADA-SCID.
Stirnadel-Farrant H, Kudari M, Garman N, Imrie J, Chopra B, Giannelli S, Gabaldo M, Corti A, Zancan S, Aiuti A, Cicalese MP, Batta R, Appleby J, Davinelli M, Ng P. Stirnadel-Farrant H, et al. Among authors: kudari m. Orphanet J Rare Dis. 2018 Apr 6;13(1):49. doi: 10.1186/s13023-018-0791-9. Orphanet J Rare Dis. 2018. PMID: 29625577 Free PMC article.
Lessons from The Netherlands.
Kwong E, Graft B, Hussain M, Kudari M, Mukhtar S, Ramanuj P. Kwong E, et al. Among authors: kudari m. Health Serv J. 2013 Oct 11;123(6370):20. Health Serv J. 2013. PMID: 24371950 No abstract available.