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

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

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

Lentiviral gene therapy for X-linked chronic granulomatous disease

慢病毒基因疗法治疗X连锁慢性肉芽肿病

Kohn, Donald B; Booth, Claire; Kang, Elizabeth M; Pai, Sung-Yun; Shaw, Kit L; Santilli, Giorgia; Armant, Myriam; Buckland, Karen F; Choi, Uimook; De Ravin, Suk See; Dorsey, Morna J; Kuo, Caroline Y; Leon-Rico, Diego; Rivat, Christine; Izotova, Natalia; Gilmour, Kimberly; Snell, Katie; Dip, Jinhua Xu-Bayford; Darwish, Jinan; Morris, Emma C; Terrazas, Dayna; Wang, Leo D; Bauser, Christopher A; Paprotka, Tobias; Kuhns, Douglas B; Gregg, John; Raymond, Hayley E; Everett, John K; Honnet, Geraldine; Biasco, Luca; Newburger, Peter E; Bushman, Frederic D; Grez, Manuel; Gaspar, H Bobby; Williams, David A; Malech, Harry L; Galy, Anne; Thrasher, Adrian J

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 #

The downregulated membrane expression of CD18 in CD34+ cells defines a primitive population of human hematopoietic stem cells

CD34+细胞中CD18膜表达下调定义了人类造血干细胞的原始群体。

Cristina Mesa-Núñez ,Diego Leon-Rico ,Montserrat Aldea ,Carlos Damián ,Raquel Sanchez-Baltasar ,Rebeca Sanchez ,Omaira Alberquilla ,José Carlos Segovia ,Juan Antonio Bueren ,Elena Almarza

Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease

慢病毒造血干细胞基因治疗挽救庞贝病小鼠模型的临床表型

Giuseppa Piras, Claudia Montiel-Equihua, Yee-Ka Agnes Chan, Slawomir Wantuch, Daniel Stuckey, Derek Burke, Helen Prunty, Rahul Phadke, Darren Chambers, Armando Partida-Gaytan, Diego Leon-Rico, Neelam Panchal, Kathryn Whitmore, Miguel Calero, Sara Benedetti, Giorgia Santilli, Adrian J Thrasher, H Bob

Lentiviral Vector-Mediated Correction of a Mouse Model of Leukocyte Adhesion Deficiency Type I

利用慢病毒载体介导的I型白细胞黏附缺陷小鼠模型矫正

Leon-Rico, Diego; Aldea, Montserrat; Sanchez-Baltasar, Raquel; Mesa-Nuñez, Cristina; Record, Julien; Burns, Siobhan O; Santilli, Giorgia; Thrasher, Adrian J; Bueren, Juan A; Almarza, Elena

Comparison of haematopoietic stem cell engraftment through the retro-orbital venous sinus and the lateral vein: alternative routes for bone marrow transplantation in mice

经眶后静脉窦和侧静脉植入造血干细胞的比较:小鼠骨髓移植的替代途径

D Leon-Rico, M Fernández-García, M Aldea, R Sánchez, M Peces-Barba, J Martinez-Palacio, R M Yáñez, E Almarza