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IL10 gene mutations do not impair Tr1 differentiation in vivo and can be functionally corrected by precise gene editing Strubbe, Steven; Boss, Allison; Wang, Bing; Mantilla, Michelle Mercedes; Freeborn, Robert Arthur; Guevara Becerra, Martha; Gharahdaghi, Nima; Uhlig, Holm H.; Porteus, Matthew Hebden; Arkwright, Peter D.; Roncarolo, Maria-Grazia; Bacchetta, Rosa 分享 收藏
Whole Exome Sequencing in Children With Autoimmune Hepatitis Identified Mutations in Genes Involved in the mTORC1 Signaling Pathway Gaigne, Lea-Philippine; Besnard, Caroline; Debeaupuis, Orianne; Degtiar, Artem; Nhat, Duong Ho; Stolzenberg, Marie-Claude; Camara, Fatou; Pelle, Olivier; Schvartz, Adrien; Perin, Melodie; Almes, Marion; Darmellah-Remil, Amaria; Gonzales, Emmanuel; Gardin, Antoine; Habes, Dalila; Destombe, Sylvie; De Martin, Eleonora; Duclos-Vallee, Jean-Charles; Arkwright, Peter D.; Rieux-Laucat, Frederic; Jacquemin, Emmanuel; Magerus, Aude 分享 收藏
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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
The effect of dupilumab on caregiver- and patient-reported outcomes in young children with moderate-to-severe atopic dermatitis: Results from a placebo- controlled, phase 3 study Paller, Amy S.; Silverberg, Jonathan I.; Simpson, Eric L.; Cork, Michael J.; Arkwright, Peter D.; Chen, Zhen; Bansal, Ashish; Prescilla, Randy; Wang, Zhixiao; Marco, Ainara R. 分享 收藏
Inborn errors of immunity reveal molecular requirements for generation and maintenance of human CD4+ IL-9-expressing cells 原发性免疫缺陷揭示了产生和维持人类CD4+ IL-9表达细胞的分子需求 Rao, Geetha; Mack, Corinne D.; Nguyen, Tina; Wong, Natalie; Payne, Kathryn; Worley, Lisa; Gray, Paul E.; Wong, Melanie; Hsu, Peter; Stormon, Michael O.; Preece, Kahn; Suan, Daniel; O'Sullivan, Michael; Blincoe, Annaliesse K.; Sinclair, Jan; Okada, Satoshi; Hambleton, Sophie; Arkwright, Peter D.; Boztug, Kaan; Stepensky, Polina; Cooper, Megan A.; Bezrodnik, Liliana; Nadeau, Kari C.; Abraham, Roshini S.; Seppanen, Mikko R. J.; Bustamante, Jacinta; Forbes, Lisa R.; Leiding, Jennifer W.; Meyts, Isabelle; Jouanguy, Emmanuelle; Boisson-Dupuis, Stephanie; Uzel, Gulbu; Tangye, Stuart G.; Ma, Cindy S. 分享 收藏
Hematopoietic Stem Cell Transplantation for C1q Deficiency: A Study on Behalf of the EBMT Inborn Errors Working Party Buso, Helena; Adam, Etai; Arkwright, Peter D.; Bhattad, Sagar; Hamidieh, Amir Ali; Behfar, Maryam; Belot, Alexandre; Benezech, Sarah; Chan, Alice Y.; Crow, Yanick J.; Dvorak, Christopher C.; Flinn, Aisling M.; Kapoor, Urvi; Lankester, Arjan; Kobayashi, Masao; Matsumura, Risa; Mottaghipisheh, Hadi; Okada, Satoshi; Ouachee, Marie; Parvaneh, Nima; Ramprakash, Stalin; Satwani, Prakash; Sharafian, Samin; Triaille, Clement; Wynn, Robert F.; Movahedi, Nasim; Ziaee, Vahid; Williams, Eleri; Slatter, Mary; Gennery, Andrew R. 分享 收藏
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Exclusive Characteristics of the p.E555K Dominant-Negative Variant in Autosomal Dominant E47 Deficiency (vol 44, 167, 2024) Utsumi, Takanori; Tsumura, Miyuki; Yashiro, Masato; Kato, Zenichiro; Noma, Kosuke; Sakura, Fumiaki; Kagawa, Reiko; Mizoguchi, Yoko; Karakawa, Shuhei; Ohnishi, Hidenori; Cunningham-Rundles, Charlotte; Arkwright, Peter D.; Kobayashi, Masao; Kanegane, Hirokazu; Bogunovic, Dusan; Boisson, Bertrand; Casanova, Jean-Laurent; Asano, Takaki; Okada, Satoshi 分享 收藏
Dupilumab efficacy and safety up to 2 years in children aged 6 months to 5 years with atopic dermatitis Dupilumab在6个月至5岁特应性皮炎儿童中长达2年的疗效和安全性 Paller, Amy S.; Simpson, Eric L.; Siegfried, Elaine C.; Cork, Michael J.; Arkwright, Peter D.; Pinter, Andreas; Dubost-Brama, Ariane; Laws, Elizabeth; Chen, Zhen; Bansal, Ashish; Prescilla, Randy; Nguyen, Tien, V 分享 收藏
Exclusive Characteristics of the p.E555K Dominant-Negative Variant in Autosomal Dominant E47 Deficiency Utsumi, Takanori; Tsumura, Miyuki; Yashiro, Masato; Kato, Zenichiro; Noma, Kosuke; Sakura, Fumiaki; Kagawa, Reiko; Mizoguchi, Yoko; Karakawa, Shuhei; Ohnishi, Hidenori; Cunningham-Rundles, Charlotte; Arkwright, Peter D.; Kobayashi, Masao; Kanegane, Hirokazu; Bogunovic, Dusan; Boisson, Bertrand; Casanova, Jean-Laurent; Asano, Takaki; Okada, Satoshi 分享 收藏
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Omenn Syndrome in Two Infants with Different Hypomorphic Variants in Janus Kinase 3 Tsilifis, Christo; Spegarova, Jarmila Stremenova; Good, Ross; Griffin, Helen; Engelhardt, Karin R.; Graham, Sophie; Hughes, Stephen; Arkwright, Peter D.; Hambleton, Sophie; Gennery, Andrew R. 分享 收藏
Detection of signature double-negative T cells is a predictive marker to identify autoimmune lymphoproliferative syndrome associated with FAS loss of function Eisenhauer, Nina; Miano, Maurizio; Naumann-Bartsch, Nora; Leyh, Joerg; Dell'Orso, Gianluca; Aigner, Michael; Fecker, Gisela; Hinze, Claas; Wittkowski, Helmut; Bruns, Heiko; Zierk, Jakob; Metzler, Markus; Arkwright, Peter D.; Graw, Frederik; Mackensen, Andreas; Voelkl, Simon 分享 收藏
Infections in Children Aged 6 Months to 5 Years Treated with Dupilumab in a Placebo-Controlled Clinical Trial of Moderate-to-Severe Atopic Dermatitis Paller, Amy S.; Siegfried, Elaine C.; Cork, Michael J.; Arkwright, Peter D.; Eichenfield, Lawrence F.; Ramien, Michele; Khokhar, Faisal A.; Chen, Zhen; Zhang, Annie; Cyr, Sonya L. 分享 收藏
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