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

TANGO2-related rhabdomyolysis symptoms are associated with abnormal autophagy functioning

TANGO2 相关横纹肌溶解症症状与自噬功能异常有关

Hortense de Calbiac #, Sebastian Montealegre #, Marjolène Straube, Solène Renault, Hugo Debruge, Loïc Chentout, Sorana Ciura, Apolline Imbard, Edouard Le Guillou, Anca Marian, Nicolas Goudin, Laure Caccavelli, Sylvie Fabrega, Arnaud Hubas, Peter van Endert, Nicolas Dupont, Julien Diana, Edor Kabashi

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

基因治疗患者的克隆追踪揭示了人类造血分化程序的多样性

Six, Emmanuelle; Guilloux, Agathe; Denis, Adeline; Lecoules, Arnaud; Magnani, Alessandra; Vilette, Romain; Male, Frances; Cagnard, Nicolas; Delville, Marianne; Magrin, Elisa; Caccavelli, Laure; Roudaut, Cécile; Plantier, Clemence; Sobrino, Steicy; Gregg, John; Nobles, Christopher L; Everett, John K; Hacein-Bey-Abina, Salima; Galy, Anne; Fischer, Alain; Thrasher, Adrian J; André, Isabelle; Cavazzana, Marina; Bushman, Frederic D

INSPIIRED: Quantification and Visualization Tools for Analyzing Integration Site Distributions

INSPIIRED:用于分析整合位点分布的量化和可视化工具

Berry, Charles C; Nobles, Christopher; Six, Emmanuelle; Wu, Yinghua; Malani, Nirav; Sherman, Eric; Dryga, Anatoly; Everett, John K; Male, Frances; Bailey, Aubrey; Bittinger, Kyle; Drake, Mary J; Caccavelli, Laure; Bates, Paul; Hacein-Bey-Abina, Salima; Cavazzana, Marina; Bushman, Frederic D

INSPIIRED: A Pipeline for Quantitative Analysis of Sites of New DNA Integration in Cellular Genomes

INSPIIRED:用于定量分析细胞基因组中新DNA整合位点的流程

Sherman, Eric; Nobles, Christopher; Berry, Charles C; Six, Emmanuelle; Wu, Yinghua; Dryga, Anatoly; Malani, Nirav; Male, Frances; Reddy, Shantan; Bailey, Aubrey; Bittinger, Kyle; Everett, John K; Caccavelli, Laure; Drake, Mary J; Bates, Paul; Hacein-Bey-Abina, Salima; Cavazzana, Marina; Bushman, Frederic D

Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome

基因治疗对重症Wiskott-Aldrich综合征患者的疗效

Hacein-Bey Abina, Salima; Gaspar, H Bobby; Blondeau, Johanna; Caccavelli, Laure; Charrier, Sabine; Buckland, Karen; Picard, Capucine; Six, Emmanuelle; Himoudi, Nourredine; Gilmour, Kimberly; McNicol, Anne-Marie; Hara, Havinder; Xu-Bayford, Jinhua; Rivat, Christine; Touzot, Fabien; Mavilio, Fulvio; Lim, Annick; Treluyer, Jean-Marc; Héritier, Sébastien; Lefrère, Francois; Magalon, Jeremy; Pengue-Koyi, Isabelle; Honnet, Géraldine; Blanche, Stéphane; Sherman, Eric A; Male, Frances; Berry, Charles; Malani, Nirav; Bushman, Frederic D; Fischer, Alain; Thrasher, Adrian J; Galy, Anne; Cavazzana, Marina

A modified γ-retrovirus vector for X-linked severe combined immunodeficiency

用于治疗X连锁重症联合免疫缺陷的改良型γ逆转录病毒载体

Hacein-Bey-Abina, Salima; Pai, Sung-Yun; Gaspar, H Bobby; Armant, Myriam; Berry, Charles C; Blanche, Stephane; Bleesing, Jack; Blondeau, Johanna; de Boer, Helen; Buckland, Karen F; Caccavelli, Laure; Cros, Guilhem; De Oliveira, Satiro; Fernández, Karen S; Guo, Dongjing; Harris, Chad E; Hopkins, Gregory; Lehmann, Leslie E; Lim, Annick; London, Wendy B; van der Loo, Johannes C M; Malani, Nirav; Male, Frances; Malik, Punam; Marinovic, M Angélica; McNicol, Anne-Marie; Moshous, Despina; Neven, Benedicte; Oleastro, Matías; Picard, Capucine; Ritz, Jerome; Rivat, Christine; Schambach, Axel; Shaw, Kit L; Sherman, Eric A; Silberstein, Leslie E; Six, Emmanuelle; Touzot, Fabien; Tsytsykova, Alla; Xu-Bayford, Jinhua; Baum, Christopher; Bushman, Frederic D; Fischer, Alain; Kohn, Donald B; Filipovich, Alexandra H; Notarangelo, Luigi D; Cavazzana, Marina; Williams, David A; Thrasher, Adrian J

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

人类β-地中海贫血基因治疗后的输血依赖性和HMGA2激活

Marina Cavazzana-Calvo, Emmanuel Payen, Olivier Negre, Gary Wang, Kathleen Hehir, Floriane Fusil, Julian Down, Maria Denaro, Troy Brady, Karen Westerman, Resy Cavallesco, Beatrix Gillet-Legrand, Laure Caccavelli, Riccardo Sgarra, Leila Maouche-Chrétien, Françoise Bernaudin, Robert Girot, Ronald Dora

Relationship between regulatory T cells and immune activation in human immunodeficiency virus-infected patients interrupting antiretroviral therapy

中断抗逆转录病毒治疗的人类免疫缺陷病毒感染者调节性T细胞与免疫激活的关系

Laurence Weiss, Christophe Piketty, Lambert Assoumou, Céline Didier, Laure Caccavelli, Vladimira Donkova-Petrini, Yves Levy, Pierre-Marie Girard, Marianne Burgard, Jean-Paul Viard, Christine Rouzioux, Dominique Costagliola; ANRS 116 SALTO study group

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

SCID-X1逆转录病毒介导的基因治疗后4例患者发生插入性肿瘤发生

Salima Hacein-Bey-Abina,Alexandrine Garrigue, Gary P Wang, Jean Soulier, Annick Lim, Estelle Morillon, Emmanuelle Clappier, Laure Caccavelli, Eric Delabesse, Kheira Beldjord, Vahid Asnafi, Elizabeth MacIntyre, Liliane Dal Cortivo, Isabelle Radford, Nicole Brousse, François Sigaux, Despina Moshous, Julia Hauer, Arndt Borkhardt, Bernd H Belohradsky, Uwe Wintergerst, Maria C Velez, Lily Leiva, Ricardo Sorensen, Nicolas Wulffraat, Stéphane Blanche, Frederic D Bushman, Alain Fischer, Marina Cavazzana-Calvo