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

Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy

体外修饰造血干细胞和祖细胞用于基因治疗

Williams, David A; Kohn, Donald B; Thrasher, Adrian J

Tailoring capsid-directed evolution technology for improved AAV-mediated CAR-T generation.

针对衣壳定向进化技术进行改造,以改进AAV介导的CAR-T细胞生成

Westhaus Adrian, Barba-Sarasua Elena, Chen Yuyan, Hsu Kenneth, Scott Suzanne, Knight Maddison, Haase Florencia, Mesa Mora Santiago, Houghton Benjamin C, Roca-Pinilla Ramon, Kalajdzic Predrag, O'Neill Geraldine, Thrasher Adrian J, Santilli Giorgia, Lisowski Leszek

Dominant negative variants in ITPR3 impair T cell Ca2+ dynamics causing combined immunodeficiency.

ITPR3 中的显性负性变异会损害 T 细胞 Ca2+ 动力学,导致联合免疫缺陷

Blanco Elena, Camps Carme, Bahal Sameer, Kerai Mohit D, Ferla Matteo P, Rochussen Adam M, Handel Adam E, Golwala Zainab M, Spiridou Goncalves Helena, Kricke Susanne, Klein Fabian, Zhang Fang, Zinghirino Federica, Evans Grace, Keane Thomas M, Lizot Sabrina, Kusters Maaike A A, Iro Mildred A, Patel Sanjay V, Morris Emma C, Burns Siobhan O, Radcliffe Ruth, Vasudevan Pradeep, Price Arthur, Gillham Olivia, Valdebenito Gabriel E, Stewart Grant S, Worth Austen, Adams Stuart P, Duchen Michael, André Isabelle, Adams David J, Santili Giorgia, Gilmour Kimberly C, Holländer Georg A, Davies E Graham, Taylor Jenny C, Griffiths Gillian M, Thrasher Adrian J, Dhalla Fatima, Kreins Alexandra Y

Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice

慢病毒基因疗法治疗腺苷脱氨酶2缺乏症(DADA2)的临床前评估:在人源化NBSGW小鼠中的移植效率和生物分布

Hong, Ying; Burleigh, Alice; Liao, Aiyin; Yeung, Jenny; Bian, Yixin; Sebire, Neil; Ogunbiyi, Olumide; Omoyinmi, Ebun; Thrasher, Adrian J; Morris, Emma; Brogan, Paul A; Eleftheriou, Despina

Preclinical ex vivo IL2RG gene therapy using autologous hematopoietic stem cells as an effective and safe treatment for X-linked severe combined immunodeficiency disease

利用自体造血干细胞进行体外IL2RG基因治疗的临床前研究,表明其可作为一种有效且安全的X连锁重症联合免疫缺陷病治疗方法。

Hu, Mingfeng; Xu, Qiling; Zhang, Fang; Buckland, Karen F; Gao, Yelei; Du, Weixia; Ding, Yuan; Zhou, Lina; Sun, Xiulian; Ma, Lijia; Zhang, Zhiyong; Tang, Xuemei; Zhao, Xiaodong; Thrasher, Adrian J; An, Yunfei

Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy

慢病毒基因疗法治疗脑型肾上腺脑白质营养不良

Eichler, Florian; Duncan, Christine N; Musolino, Patricia L; Lund, Troy C; Gupta, Ashish O; De Oliveira, Satiro; Thrasher, Adrian J; Aubourg, Patrick; Kühl, Jörn-Sven; Loes, Daniel J; Amartino, Hernan; Smith, Nicholas; Folloni Fernandes, Juliana; Sevin, Caroline; Sankar, Raman; Hussain, Shaun A; Gissen, Paul; Dalle, Jean-Hugues; Platzbecker, Uwe; Downey, Gerald F; McNeil, Elizabeth; Demopoulos, Laura; Dietz, Andrew C; Thakar, Himal L; Orchard, Paul J; Williams, David A

Valproic Acid Confers Functional Pluripotency to Human Amniotic Fluid Stem Cells in a Transgene-free Approach

丙戊酸以非转基因方式赋予人羊水干细胞功能性多能性

Moschidou, Dafni; Mukherjee, Sayandip; Blundell, Michael P; Drews, Katharina; Jones, Gemma N; Abdulrazzak, Hassan; Nowakowska, Beata; Phoolchund, Anju; Lay, Kenneth; Ramasamy, T Selvee; Cananzi, Mara; Nettersheim, Daniel; Sullivan, Mark; Frost, Jennifer; Moore, Gudrun; Vermeesch, Joris R; Fisk, Nicholas M; Thrasher, Adrian J; Atala, Anthony; Adjaye, James; Schorle, Hubert; De Coppi, Paolo; Guillot, Pascale V

Anti-CD45 PBD-based antibody-drug conjugates are effective targeted conditioning agents for gene therapy and stem cell transplant

基于抗CD45 PBD的抗体药物偶联物是基因治疗和干细胞移植的有效靶向预处理剂。

Yeung, Jenny; Liao, Aiyin; Shaw, Matthew; Silva, Soraia; Vetharoy, Winston; Rico, Diego Leon; Kirby, Ian; Zammarchi, Francesca; Havenith, Karin; de Haan, Lolke; van Berkel, Patrick H; Sebire, Neil; Ogunbiyi, Olumide K; Booth, Claire; Gaspar, H Bobby; Thrasher, Adrian J; Chester, Kerry A; Amrolia, Persis J

CRISPR/Cas9-Based Disease Modeling and Functional Correction of Interleukin 7 Receptor Alpha Severe Combined Immunodeficiency in T-Lymphocytes and Hematopoietic Stem Cells

基于 CRISPR/Cas9 的白细胞介素 7 受体 α 重症联合免疫缺陷疾病建模及功能矫正(T 淋巴细胞和造血干细胞)

Rai, Rajeev; Steinberg, Zohar; Romito, Marianna; Zinghirino, Federica; Hu, Yi-Ting; White, Nathan; Naseem, Asma; Thrasher, Adrian J; Turchiano, Giandomenico; Cavazza, Alessia

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency

自体体外慢病毒基因疗法治疗腺苷脱氨酶缺乏症

Kohn, Donald B; Booth, Claire; Shaw, Kit L; Xu-Bayford, Jinhua; Garabedian, Elizabeth; Trevisan, Valentina; Carbonaro-Sarracino, Denise A; Soni, Kajal; Terrazas, Dayna; Snell, Katie; Ikeda, Alan; Leon-Rico, Diego; Moore, Theodore B; Buckland, Karen F; Shah, Ami J; Gilmour, Kimberly C; De Oliveira, Satiro; Rivat, Christine; Crooks, Gay M; Izotova, Natalia; Tse, John; Adams, Stuart; Shupien, Sally; Ricketts, Hilory; Davila, Alejandra; Uzowuru, Chilenwa; Icreverzi, Amalia; Barman, Provaboti; Campo Fernandez, Beatriz; Hollis, Roger P; Coronel, Maritess; Yu, Allen; Chun, Krista M; Casas, Christian E; Zhang, Ruixue; Arduini, Serena; Lynn, Frances; Kudari, Mahesh; Spezzi, Andrea; Zahn, Marco; Heimke, Rene; Labik, Ivan; Parrott, Roberta; Buckley, Rebecca H; Reeves, Lilith; Cornetta, Kenneth; Sokolic, Robert; Hershfield, Michael; Schmidt, Manfred; Candotti, Fabio; Malech, Harry L; Thrasher, Adrian J; Gaspar, H Bobby