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收藏Outcomes of Hematopoietic Stem Cell Transplantation in 5 Patients with Autosomal Recessive RIPK1-Deficiency造血干细胞移植治疗5例常染色体隐性RIPK1-Deficiency的疗效分析
Walsh, Rebecca B.; Mcnaughton, Peter; Nademi, Zohreh; Laberko, Alexandra; Balashov, Dmitry; Al-Mousa, Hamoud; Arkwright, Peter D.; Wynn, Robert F.; Flood, Terry; Williams, Eleri; Cant, Andrew; Abinun, Mario; Hambleton, Sophie; Slatter, Mary; Gennery, Andrew R.; Lum, Su Han; Owens, Stephen 分享 收藏 分享 收藏 Hematopoietic cell transplantation in severe combined immunodeficiency: The SCETIDE 2006-2014 European cohort严重联合免疫缺陷的造血细胞移植: SCETIDE 2006 2014的欧洲队列Lankester, Arjan C.; Neven, Benedicte; Mahlaoui, Nizar; von Asmuth, Erik G. J.; Courteille, Virginie; Alligon, Mikael; Albert, Michael H.; Serra, Isabelle Badell; Bader, Peter; Balashov, Dmitry; Beier, Rita; Bertrand, Yves; Blanche, Stephane; Bordon, Victoria; Bredius, Robbert G.; Cant, Andrew; Cavazzana, Marina; Diaz-de-Heredia, Cristina; Dogu, Figen; Ehlert, Karoline; Entz-Werle, Natacha; Fasth, Anders; Ferrua, Francesca; Ferster, Alina; Formankova, Renata; Friedrich, Wilhelm; Gonzalez-Vicent, Marta; Gozdzik, Jolanta; Gungor, Tayfun; Hoenig, Manfred; Ikinciogullari, Aydan; Kalwak, Krzysztof; Kansoy, Savas; Kupesiz, Alphan; Lanfranchi, Arnalda; Lindemans, Caroline A.; Meisel, Roland; Michel, Gerard; Miranda, Nuno A. A.; Moraleda, Jose; Moshous, Despina; Pichler, Herbert; Rao, Kanchan; Sedlacek, Petr; Slatter, Mary; Soncini, Elena; Speckmann, Carsten; Sundin, Mikael; Toren, Amos; Vettenranta, Kim; Worth, Austen; Yesilipek, Mehmet A.; Zecca, Marco; Porta, Fulvio; Schulz, Ansgar; Veys, Paul; Fischer, Alain; Gennery, Andrew R. 分享 收藏 TCRαβ-Depleted Haploidentical Grafts Are a Safe Alternative to HLA-Matched Unrelated Donor Stem Cell Transplants for Infants with Severe Combined ImmunodeficiencyTsilifis, Christo; Lum, Su Han; Nademi, Zohreh; Hambleton, Sophie; Flood, Terence J.; Williams, Eleri J.; Owens, Stephen; Abinun, Mario; Cant, Andrew J.; Slatter, Mary A.; Gennery, Andrew R. 分享 收藏 T-replete HLA-matched grafts vs T-depleted HLA-mismatched grafts in inborn errors of immunityLum, Su Han; Greener, Sinead; Perez-Heras, Inigo; Drozdov, Daniel; Payne, Rebecca P.; Watson, Helen; Carruthers, Kay; January, Robert; Nademi, Zohreh; Owens, Stephen; Williams, Eleri; Waugh, Sheila; Burton-Fanning, Shirelle; Leahy, Timmothy Ronan; Cant, Andrew; Abinun, Mario; Flood, Terry; Hambleton, Sophie; Gennery, Andrew R.; Slatter, Mary 分享 收藏 Hematopoietic Cell Transplantation Ameliorates Autoinflammation in A20 Haploinsufficiency造血细胞移植可改善A20单倍体不足的自身炎症Shiraki, Mayuka; Williams, Eleri; Yokoyama, Norifumi; Shinoda, Kunihiro; Nademi, Zohreh; Matsumoto, Kana; Nihira, Hiroshi; Honda, Yoshitaka; Izawa, Kazushi; Nishikomori, Ryuta; Slatter, Mary A.; Cant, Andrew J.; Gennery, Andrew R.; Ohnishi, Hidenori; Kanegane, Hirokazu 分享 收藏 Variants in STXBP3 are Associated with Very Early Onset Inflammatory Bowel Disease, Bilateral Sensorineural Hearing Loss and Immune DysregulationOuahed, Jodie; Kelsen, Judith R.; Spessott, Waldo A.; Kooshesh, Kameron; Sanmillan, Maria L.; Dawany, Noor; Sullivan, Kathleen E.; Hamilton, Kathryn E.; Slowik, Voytek; Nejentsev, Sergey; Neves, Joao Farela; Flores, Helena; Chung, Wendy K.; Wilson, Ashley; Anyane-Yeboa, Kwame; Wou, Karen; Jain, Preti; Field, Michael; Tollefson, Sophia; Dent, Maiah H.; Li, Dalin; Naito, Takeo; McGovern, Dermot P. B.; Kwong, Andrew C.; Taliaferro, Faith; Ordovas-Montanes, Jose; Horwitz, Bruce H.; Kotlarz, Daniel; Klein, Christoph; Evans, Jonathan; Dorsey, Jill; Warner, Neil; Elkadri, Abdul; Muise, Aleixo M.; Goldsmith, Jeffrey; Thompson, Benjamin; Engelhardt, Karin R.; Cant, Andrew J.; Hambleton, Sophie; Barclay, Andrew; Toth-Petroczy, Agnes; Vuzman, Dana; Carmichael, Nikkola; Bodea, Corneliu; Cassa, Christopher A.; Devoto, Marcella; Maas, Richard L.; Behrens, Edward M.; Giraudo, Claudio G.; Snapper, Scott B. 分享 收藏 Hematopoietic Stem Cell Transplantation Resolves the Immune Deficit Associated with STAT3-Dominant-Negative Hyper-IgE Syndrome造血干细胞移植可解决与STAT3-Dominant-Negative高IgE综合征相关的免疫缺陷Harrison, Stephanie C.; Tsilifis, Christo; Slatter, Mary A.; Nademi, Zohreh; Worth, Austen; Veys, Paul; Ponsford, Mark J.; Jolles, Stephen; Al-Herz, Waleed; Flood, Terence; Cant, Andrew J.; Doffinger, Rainer; Barcenas-Morales, Gabriela; Carpenter, Ben; Hough, Rachael; Haraldsson, Asgeir; Heimall, Jennifer; Grimbacher, Bodo; Abinun, Mario; Gennery, Andrew R. 分享 收藏 Outcome of autoimmune cytopenia after hematopoietic cell transplantation in primary immunodeficiencyLum, Su Han; Selvarajah, Sabeena; Deya-Martinez, Angela; McNaughton, Peter; Sobh, Ali; Waugh, Sheila; Burton-Fanning, Shirelle; Newton, Lisa; Gandy, Julie; Nademi, Zohreh; Owens, Stephen; Williams, Eleri; Emonts, Marieke; Flood, Terry; Cant, Andrew; Abinun, Mario; Hambleton, Sophie; R. Gennery, Andrew; Slatter, Mary 分享 收藏 New insights into risk factors for transplant-associated thrombotic microangiopathy in pediatric HSCTElfeky, Reem; Lucchini, Giovanna; Lum, Su-Han; Ottaviano, Giorgio; Builes, Natalia; Nademi, Zohreh; Battersby, Alexandra; Flood, Terence; Owens, Stephen; Cant, Andrew J.; Young, Helen; Greener, Sinead; Walsh, Patrick; Kavanagh, David; Annavarapu, Srinivas; Rao, Kanchan; Amrolia, Persis; Chiesa, Robert; Worth, Austen; Booth, Claire; Skinner, Roderick; Doncheva, Bilyana; Standing, Joseph; Gennery, Andrew R.; Qasim, Waseem; Slatter, Mary; Veys, Paul 分享 收藏 分享 收藏 Proposed Therapeutic Range of Treosulfan in Reduced Toxicity Pediatric Allogeneic Hematopoietic Stem Cell Transplant Conditioning: Results From a Prospective TrialChiesa, Robert; Standing, Joseph F.; Winter, Robert; Nademi, Zohreh; Chu, Jan; Pinner, Danielle; Kloprogge, Frank; McLellen, Susan; Amrolia, Persis J.; Rao, Kanchan; Lucchini, Giovanna; Silva, Juliana; Ciocarlie, Oana; Lazareva, Arina; Gennery, Andrew R.; Doncheva, Bilyana; Cant, Andrew J.; Hambleton, Sophie; Flood, Terence; Rogerson, Elizabeth; Devine, Kirsty; Prunty, Helen; Heales, Simon; Veys, Paul; Slatter, Mary 分享 收藏 Novel Gain-of-Function Mutation in Stat1 Sumoylation Site Leads to CMC/CID Phenotype Responsive to RuxolitinibAl Shehri, Tariq; Gilmour, Kimberly; Gothe, Florian; Loughlin, Sam; Bibi, Shahnaz; Rowan, Andrew D.; Grainger, Angela; Mohanadas, Thivytra; Cant, Andrew J.; Slatter, Mary A.; Hambleton, Sophie; Lilic, Desa; Leahy, Timothy R. 分享 收藏 The European Society for Immunodeficiencies (ESID) Registry Working Definitions for the Clinical Diagnosis of Inborn Errors of Immunity欧洲免疫缺陷学会 (ESID) 注册用于先天性免疫缺陷临床诊断的工作定义Seidel, Markus G.; Kindle, Gerhard; Gathmann, Benjamin; Quinti, Isabella; Buckland, Matthew; van Montfrans, Joris; Scheible, Raphael; Ruschb, Stephan; Gasteiger, Lukas M.; Grimbacher, Bodo; Mahlaoui, Nizar; Ehl, Stephan; Abinun, Mario; Albert, Michael; Cohen, Sarah Beaussant; Bustamante, Jacinta; Cant, Andrew; Casanova, Jean-Laurent; Chapel, Helen; de Saint Basile, Genevieve; de Vries, Esther; Dokal, Inderjeet; Donadieu, Jean; Durandy, Anne; Edgar, David; Espanol, Teresa; Etzioni, Amos; Fischer, Alain; Gaspar, Bobby; Gatti, Richard; Gennery, Andrew; Grigoriadou, Sofia; Holland, Steven; Janka, Gritta; Kanariou, Maria; Klein, Christoph; Lachmann, Helen; Lilic, Desa; Manson, Ania; Martinez, Natalia; Meyts, Isabelle; Moes, Nicolette; Moshous, Despina; Neven, Benedicte; Ochs, Hans; Picard, Capucine; Renner, Ellen; Rieux-Laucat, Frederic; Seger, Reinhard; Soresina, Annarosa; Stoppa-Lyonnet, Dominique; Thon, Vojtech; Thrasher, Adrian; van de Veer-Donk, Frank; Villa, Anna; Weemaes, Corry; Warnatz, Klaus; Wolska, Beata; Zhang, Shen-Yin 分享 收藏 New graft manipulation strategies improve the outcome of mismatched stem cell transplantation in children with primary immunodeficienciesElfeky, Reem; Shah, Ravi M.; Unni, Mohamed N. M.; Ottaviano, Giorgio; Rao, Kanchan; Chiesa, Robert; Amrolia, Persis; Worth, Austen; Flood, Terry; Abinun, Mario; Hambleton, Sophie; Cant, Andrew J.; Gilmour, Kimberly; Adams, Stuart; Ahsan, Gul; Barge, Dawn; Gennery, Andrew R.; Qasim, Waseem; Slatter, Mary; Veys, Paul 分享 收藏 Hematopoietic stem cell transplant effectively rescues lymphocyte differentiation and function in DOCK8-deficient patientsPillay, Bethany A.; Avery, Danielle T.; Smart, Joanne M.; Cole, Theresa; Choo, Sharon; Chan, Damien; Gray, Paul E.; Frith, Katie; Mitchell, Richard; Tri Giang Phan; Wong, Melanie; Campbell, Dianne E.; Hsu, Peter; Ziegler, John B.; Peake, Jane; Alvaro, Frank; Picard, Capucine; Bustamante, Jacinta; Neven, Benedicte; Cant, Andrew J.; Uzel, Gulbu; Arkwright, Peter D.; Casanova, Jean-Laurent; Su, Helen C.; Freeman, Alexandra F.; Shah, Nirali; Hickstein, Dennis D.; Tangye, Stuart G.; Ma, Cindy S. 分享 收藏 Prioritizing research challenges and funding for allergy and asthma and the need for translational research-The European Strategic Forum on Allergic Diseases优先考虑过敏和哮喘的研究挑战和资金以及转化研究的需求-欧洲过敏性疾病战略论坛Agache, Ioana; Annesi-Maesano, Isabella; Bonertz, Andreas; Branca, Francesco; Cant, Andrew; Fras, Zlatko; Ingenrieth, Frank; Namazova-Baranova, Leyla; Odemyr, Mikaela; Spanevello, Antonio; Vieths, Stefan; Yorgancioglu, Arzu; Alvaro-Lozano, Montserat; Hernandez, Domingo Barber; Chivato, Tomas; Del Giacco, Stefano; Diamant, Zuzana; Eguiluz-Gracia, Ibon; van Wijk, Roy Gert; Gevaert, Philippe; Graessel, Anke; Hellings, Peter; Hoffmann-Sommergruber, Karin; Jutel, Marek; Lau, Susanne; Lauerma, Antti; Olaguibel, Jose Maria; O'Mahony, Liam; Ozdemir, Cevdet; Palomares, Oscar; Pfaar, Oliver; Sastre, Joaquin; Scadding, Glennis; Schmidt-Weber, Carsten; Schmid-Grendelmeier, Peter; Shamji, Mohamed; Skypala, Isabel; Spinola, Monica; Spranger, Otto; Torres, Maria; Vereda, Andrea; Bonini, Sergio 分享 收藏 Immunodeficiency, autoimmune thrombocytopenia and enterocolitis caused by autosomal recessive deficiency of PIK3CD-encoded phosphoinositide 3-kinase δSwan, David J.; Aschenbrenner, Dominik; Lamb, Christopher A.; Chakraborty, Krishnendu; Clark, Jonathan; Pandey, Sumeet; Engelhardt, Karin R.; Chen, Rui; Cavounidis, Athena; Ding, Yuchun; Krasnogor, Natalio; Carey, Christopher D.; Acres, Meghan; Needham, Stephanie; Cant, Andrew J.; Arkwright, Peter D.; Chandra, Anita; Okkenhaug, Klaus; Uhlig, Holm H.; Hambleton, Sophie 分享 收藏 Combined liver and hematopoietic stem cell transplantation in patients with X-linked hyper-IgM syndromeBucciol, Giorgia; Nicholas, Sarah K.; Calvo, Pier Luigi; Cant, Andrew; Edgar, J. David M.; Espanol, Teresa; Ferrua, Francesca; Galicchio, Miguel; Gennery, Andrew R.; Hadzic, Nedim; Hanson, I. Celine; Kusminsky, Gustavo; Lange, Andrzej; Lanternier, Fanny; Mahlaoui, Nizar; Moshous, Despina; Nademi, Zohreh; Neven, Benedicte; Oleastro, Matias; Porta, Fulvio; Quarello, Paola; Silva, Marcelo; Slatter, Mary A.; Soncini, Elena; Stefanowicz, Marek; Tandoi, Francesco; Teisseyre, Mikolaj; Torgerson, Troy R.; Veys, Paul; Weinacht, Katja G.; Wolska-Kusnierz, Beata; Pirenne, Jacques; de la Morena, M. Teresa; Meyts, Isabelle 分享 收藏 
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