日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Safety and efficacy of gene therapy for RAG1-deficient SCID

RAG1缺陷型重症联合免疫缺陷症基因治疗的安全性和有效性

Staal, Frank J T; Pike-Overzet, Karin; de Kivit, Sander; Ott de Bruin, Lisa; Mamede, Lucia; Pergent, Martine; Prevot, Johan; Rothe, Michael; Schambach, Axel; Lankester, Arjan

Immunopathological and microbial signatures of inflammatory bowel disease in partial RAG deficiency.

部分 RAG 缺乏症炎症性肠病的免疫病理学和微生物学特征

Castagnoli Riccardo, Pala Francesca, Subramanian Poorani, Oguz Cihan, Schwarz Benjamin, Lim Ai Ing, Burns Andrew S, Fontana Elena, Bosticardo Marita, Corsino Cristina, Angelova Angelina, Delmonte Ottavia M, Kenney Heather, Riley Deanna, Smith Grace, Ott de Bruin Lisa, Oikonomou Vasileios, Dos Santos Dias Lucas, Fink Danielle, Bohrnsen Eric, Kimzey Cole D, Marseglia Gian Luigi, Alva-Lozada Guisela, Bergerson Jenna R E, Brett Ana, Brigatti Karlla W, Dimitrova Dimana, Dutmer Cullen M, Freeman Alexandra F, Ale Hanadys, Holland Steven M, Licciardi Francesco, Pasic Srdjan, Poskitt Laura E, Potts David E, Dasso Joseph F, Sharapova Svetlana O, Strauss Kevin A, Ward Brant R, Yilmaz Melis, Kuhns Douglas B, Lionakis Michail S, Daley Stephen R, Kong Heidi H, Segre Julia A, Villa Anna, Pittaluga Stefania, Walter Jolan E, Vujkovic-Cvijin Ivan, Belkaid Yasmine, Notarangelo Luigi D

RAG1 lentiviral gene therapy restores T-cell development of RAG1-SCID patient cells in artificial thymic organoids

RAG1慢病毒基因疗法可恢复RAG1-SCID患者细胞在人工胸腺类器官中的T细胞发育

Meng, Xiaolin; Melsen, Janine E; van der Holst, Rosalie; de Mooij, Bas; Vloemans, Sandra; van Eggermond, Marja; Berghuis, Dagmar; Lankester, Arjan C; de Kivit, Sander; Pike-Overzet, Karin; Langerak, Anton W; Staal, Frank J T; Ott de Bruin, Lisa M; Canté-Barrett, Kirsten

Persistent Hypogammaglobulinemia after Receiving Rituximab Post-HSCT Is Not Caused by an Intrinsic B Cell Defect

接受利妥昔单抗治疗后造血干细胞移植后持续性低丙种球蛋白血症并非由B细胞内在缺陷引起

Ott de Bruin, Lisa M; Pico-Knijnenburg, Ingrid; van Ostaijen-Ten Dam, Monique M; Weitering, Thomas J; Berghuis, Dagmar; Bredius, Robbert G M; Lankester, Arjan C; van der Burg, Mirjam

Advances in gene therapy for inborn errors of immunity

基因疗法在治疗先天性免疫缺陷方面的进展

Ott de Bruin, Lisa M; Lankester, Arjan C; Staal, Frank J T

Cysteine and hydrophobic residues in CDR3 serve as distinct T-cell self-reactivity indices

CDR3中的半胱氨酸和疏水性残基可作为不同的T细胞自身反应性指标。

Daley, Stephen R; Koay, Hui-Fern; Dobbs, Kerry; Bosticardo, Marita; Wirasinha, Rushika C; Pala, Francesca; Castagnoli, Riccardo; Rowe, Jared H; Ott de Bruin, Lisa M; Keles, Sevgi; Lee, Yu Nee; Somech, Raz; Holland, Steven M; Delmonte, Ottavia M; Draper, Debbie; Maxwell, Sandra; Niemela, Julie; Stoddard, Jennifer; Rosenzweig, Sergio D; Poliani, Pietro Luigi; Capo, Valentina; Villa, Anna; Godfrey, Dale I; Notarangelo, Luigi D

Abnormalities of T-cell receptor repertoire in CD4(+) regulatory and conventional T cells in patients with RAG mutations: Implications for autoimmunity

RAG基因突变患者CD4(+)调节性T细胞和常规T细胞中T细胞受体库的异常:对自身免疫的影响

Rowe, Jared H; Stadinski, Brian D; Henderson, Lauren A; Ott de Bruin, Lisa; Delmonte, Ottavia; Lee, Yu Nee; de la Morena, M Teresa; Goyal, Rakesh K; Hayward, Anthony; Huang, Chiung-Hui; Kanariou, Maria; King, Alejandra; Kuijpers, Taco W; Soh, Jian Yi; Neven, Benedicte; Walter, Jolan E; Huseby, Eric S; Notarangelo, Luigi D

Corrigendum: Natural Killer Cells from Patients with Recombinase-Activating Gene and Non-Homologous End Joining Gene Defects Comprise a Higher Frequency of CD56(bright) NKG2A(+++) Cells, and Yet Display Increased Degranulation and Higher Perforin Content

更正:来自重组激活基因和非同源末端连接基因缺陷患者的自然杀伤细胞中,CD56<sup>bright</sup>NKG2A<sup>+++</sup>细胞的比例更高,但同时脱颗粒作用增强,穿孔素含量也更高。

Dobbs, Kerry; Tabellini, Giovanna; Calzoni, Enrica; Patrizi, Ornella; Martinez, Paula; Giliani, Silvia Clara; Moratto, Daniele; Al-Herz, Waleed; Cancrini, Caterina; Cowan, Morton; Bleesing, Jacob; Booth, Claire; Buchbinder, David; Burns, Siobhan O; Chatila, Talal A; Chou, Janet; Daza-Cajigal, Vanessa; Ott de Bruin, Lisa M; de la Morena, Maite Teresa; Di Matteo, Gigliola; Finocchi, Andrea; Geha, Raif; Goyal, Rakesh K; Hayward, Anthony; Holland, Steven; Huang, Chiung-Hui; Kanariou, Maria G; King, Alejandra; Kaplan, Blanka; Kleva, Anastasiya; Kuijpers, Taco W; Lee, Bee Wah; Lougaris, Vassilios; Massaad, Michel; Meyts, Isabelle; Morsheimer, Megan; Neven, Benedicte; Pai, Sung-Yun; Parvaneh, Nima; Plebani, Alessandro; Prockop, Susan; Reisli, Ismail; Soh, Jian Yi; Somech, Raz; Torgerson, Troy R; Kim, Yae-Jean; Walter, Jolan E; Gennery, Andrew R; Keles, Sevgi; Manis, John P; Marcenaro, Emanuela; Moretta, Alessandro; Parolini, Silvia; Notarangelo, Luigi D

Natural Killer Cells from Patients with Recombinase-Activating Gene and Non-Homologous End Joining Gene Defects Comprise a Higher Frequency of CD56(bright) NKG2A(+++) Cells, and Yet Display Increased Degranulation and Higher Perforin Content.

来自重组激活基因和非同源末端连接基因缺陷患者的自然杀伤细胞中 CD56(bright) NKG2A(+++) 细胞的频率较高,但脱颗粒作用增强,穿孔素含量也较高

Dobbs Kerry, Tabellini Giovanna, Calzoni Enrica, Patrizi Ornella, Martinez Paula, Giliani Silvia Clara, Moratto Daniele, Al-Herz Waleed, Cancrini Caterina, Cowan Morton, Bleesing Jacob, Booth Claire, Buchbinder David, Burns Siobhan O, Chatila Talal A, Chou Janet, Daza-Cajigal Vanessa, Ott de Bruin Lisa M, de la Morena MaiteTeresa, Di Matteo Gigliola, Finocchi Andrea, Geha Raif, Goyal Rakesh K, Hayward Anthony, Holland Steven, Huang Chiung-Hui, Kanariou Maria G, King Alejandra, Kaplan Blanka, Kleva Anastasiya, Kuijpers Taco W, Lee Bee Wah, Lougaris Vassilios, Massaad Michel, Meyts Isabelle, Morsheimer Megan, Neven Benedicte, Pai Sung-Yun, Parvaneh Nima, Plebani Alessandro, Prockop Susan, Reisli Ismail, Soh Jian Yi, Somech Raz, Torgerson Troy R, Kim Yae-Jaen, Walter Jolan E, Gennery Andrew R, Keles Sevgi, Manis John P, Marcenaro Emanuela, Moretta Alessandro, Parolini Silvia, Notarangelo Luigi D

Modeling altered T-cell development with induced pluripotent stem cells from patients with RAG1-dependent immune deficiencies

利用来自 RAG1 依赖性免疫缺陷患者的诱导多能干细胞模拟 T 细胞发育异常

Brauer, Patrick M; Pessach, Itai M; Clarke, Erik; Rowe, Jared H; Ott de Bruin, Lisa; Lee, Yu Nee; Dominguez-Brauer, Carmen; Comeau, Anne M; Awong, Geneve; Felgentreff, Kerstin; Zhang, Yuhang H; Bredemeyer, Andrea; Al-Herz, Waleed; Du, Likun; Ververs, Francesca; Kennedy, Marion; Giliani, Silvia; Keller, Gordon; Sleckman, Barry P; Schatz, David G; Bushman, Frederic D; Notarangelo, Luigi D; Zúñiga-Pflücker, Juan Carlos