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

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

Hematopoietic stem cell gene editing rescues B-cell development in X-linked agammaglobulinemia

造血干细胞基因编辑挽救了X连锁无丙种球蛋白血症患者的B细胞发育

Sameer Bahal ,Marta Zinicola ,Shefta E Moula ,Thomas E Whittaker ,Andrea Schejtman ,Asma Naseem ,Elena Blanco ,Winston Vetharoy ,Yi-Ting Hu ,Rajeev Rai ,Eduardo Gomez-Castaneda ,Catarina Cunha-Santos ,Siobhan O Burns ,Emma C Morris ,Claire Booth ,Giandomenico Turchiano ,Alessia Cavazza ,Adrian J Thrasher ,Giorgia Santilli

An improved medium formulation for efficient ex vivo gene editing, expansion and engraftment of hematopoietic stem and progenitor cells

一种改进的培养基配方,用于高效体外基因编辑、扩增和植入造血干细胞和祖细胞

Rajeev Rai, Asma Naseem, Winston Vetharoy, Zohar Steinberg, Adrian J Thrasher, Giorgia Santilli, Alessia Cavazza

Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47(phox)-Deficient Chronic Granulomatous Disease

针对p47(phox)缺陷型慢性肉芽肿病的新型慢病毒基因疗法的临床前优化和安全性研究

Schejtman, Andrea; Vetharoy, Winston; Choi, Uimook; Rivat, Christine; Theobald, Narda; Piras, Giuseppa; Leon-Rico, Diego; Buckland, Karen; Armenteros-Monterroso, Elena; Benedetti, Sara; Ashworth, Michael T; Rothe, Michael; Schambach, Axel; Gaspar, Hubert Bobby; Kang, Elizabeth M; Malech, Harry L; Thrasher, Adrian J; Santilli, Giorgia

Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome

人类造血干细胞靶向基因校正治疗 Wiskott - Aldrich 综合征

Rajeev Rai, Marianna Romito, Elizabeth Rivers, Giandomenico Turchiano, Georges Blattner, Winston Vetharoy, Dariusz Ladon, Geoffroy Andrieux, Fang Zhang, Marta Zinicola, Diego Leon-Rico, Giorgia Santilli, Adrian J Thrasher #, Alessia Cavazza #

Amplification of mixed lineage leukemia gene perturbs hematopoiesis and cooperates with partial tandem duplication to induce acute myeloid leukemia

混合谱系白血病基因的扩增会扰乱造血过程,并与部分串联重复协同诱发急性髓系白血病。

Yip, Bon Ham; Tsai, Chiou Tsun; Rane, Jayant K; Vetharoy, Winston; Anguita, Eduardo; Dong, Shuo; Caligiuri, Michael A; So, Chi Wai Eric