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A

Adrian J. Thrasher

ucl great ormond street institute of child health
H指数:108
被引数:4.4万

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Clinical and biochemical correction of a patient with neurodegenerative Mucopolysaccharidosis IIIA using hematopoietic stem cell gene therapy

使用造血干细胞基因疗法对神经退行性黏多糖贮积症IIIA患者进行临床和生化纠正

Molecular Therapy
IF12
2026-09-250
PRE
Jane L. Kinsella; Simon A. Jones; Karen F. Buckland; Rebecca J. Holley; Helena Lee; Wendy Ogden; Jane Potter; Heather J. Church; Kathryn L. Brammeier; Karen L. Tylee; Stuart Ellison; Rachel Searle; Claire Booth; Farzin Farzeneh; Louise Sherwin; Adrian J. Thrasher; Stewart Rust; Brian W. Bigger; Robert F. Wynn
Unveiling the cut-and-repair cycle of designer nucleases in human stem and T cells via CLEAR-time dPCR

通过清除时间dPCR揭示人类干细胞和T细胞中设计核酸酶的切割和修复周期

Nature Communications
IF15.7
2025-11-030
OAAI
Nathan White; John Alexander Chalk; Yi-Ting Hu; Samuel Mark Pins; Chinnu Rose Joseph; Panagiotis Antoniou; Sandra Wimberger; Stina Svensson; Soraia Patricia Caetano-Silva; Anne Charlotte Adriane Mudde; Rajeev Rai; Sridhar Selvaraj; William Nelson Feist; Marianna Romito; Grzegorz Sienski; Roberto Nitsch; Claire Booth; Giorgia Santilli; Alessia Cavazza; Matthew Hebden Porteus; Marcello Maresca; Adrian James Thrasher; Giandomenico Turchiano
Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency

腺苷脱氨酶缺乏症的基因治疗长期安全性与有效性

NEW ENGLAND JOURNAL OF MEDICINE
IF78.5
2025-10-160
PRE
Booth, Claire; Masiuk, Katelyn; Vazouras, Konstantinos; Fernandes, Augustine; Xu-Bayford, Jinhua; Campo Fernandez, Beatriz; Roy, Sohini; Curio-Penny, Beatrice; Arnold, Jordyn; Terrazas, Dayna; Reid, Jack; Gilmour, Kimberly C.; Adams, Stuart; Alvarez Mediavilla, Elena; Mhaldien, Lana; O'Toole, Grainne; Ahmed, Rima; Garabedian, Elizabeth; Malech, Harry; De Ravin, Suk See; Moore, Theodore B.; De Oliveira, Satiro; Pellin, Danilo; Lin, Tsai-Yu; Dang, Thao Thi; Cornetta, Kenneth; Hershfield, Michael S.; Hara, Havinder; Thrasher, Adrian J.; Gaspar, H. Bobby; Kohn, Donald B.
Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice

腺苷脱氨酶2缺乏症(DADA2)的慢病毒基因治疗临床前评估：人源化NBSGW小鼠中的植入效率和生物分布

Gene Therapy
IF4.5
2025-06-240
OAAI
Ying Hong; Alice Burleigh; Aiyin Liao; Jenny Yeung; Yixin Bian; Neil Sebire; Olumide Ogunbiyi; Ebun Omoyinmi; Adrian J. Thrasher; Emma Morris; Paul A. Brogan; Despina Eleftheriou
Dominant negative variants in ITPR3 impair T cell Ca2+ dynamics causing combined immunodeficiency
JOURNAL OF EXPERIMENTAL MEDICINE
IF10.6
2024-11-190
OAAI
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; Andre, Isabelle; Adams, David J.; Santili, Giorgia; Gilmour, Kimberly C.; Hollaender, Georg A.; Davies, E. Graham; Taylor, Jenny C.; Griffiths, Gillian M.; Thrasher, Adrian J.; Dhalla, Fatima; Kreins, Alexandra Y.
Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial

Betibeglogene autotemcel基因治疗输血依赖性，严重基因型 β-地中海贫血 (HGB-212) 患者: 一项非随机，多中心，单臂，开放标签，单剂量，3期试验

LANCET
IF88.5
2024-11-010
PRE
Kwiatkowski, Janet L.; Walters, Mark C.; Hongeng, Suradej; Yannaki, Evangelia; Kulozik, Andreas E.; Kunz, Joachim B.; Sauer, Martin G.; Thrasher, Adrian J.; Thuret, Isabelle; Lal, Ashutosh; Tao, Ge; Ali, Shamshad; Thakar, Himal L.; Elliot, Heidi; Lodaya, Ankit; Lee, Ji; Colvin, Richard A.; Locatelli, Franco; Thompson, Alexis A.
Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy

慢病毒基因治疗脑型肾上腺脑白质营养不良

NEW ENGLAND JOURNAL OF MEDICINE
IF78.5
2024-10-104
PRE
Eichler, Florian; Duncan, Christine N.; Musolino, Patricia L.; Lund, Troy C.; Gupta, Ashish O.; De Oliveira, Satiro; Thrasher, Adrian J.; Aubourg, Patrick; Kuehl, Joern-Sven; Loes, Daniel J.; Amartino, Hernan; Smith, Nicholas; Fernandes, Juliana Folloni; 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
MOLECULAR THERAPY
IF12
2024-08-011
OA
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; V. Guillot, Pascale
Hematopoietic stem cell gene editing rescues B-cell development in X-linked agammaglobulinemia
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
IF11.2
2024-07-010
OAAI
Bahal, Sameer; Zinicola, Marta; Moula, Shefta E.; Whittaker, Thomas E.; Schejtman, Andrea; Naseem, Asma; Blanco, Elena; Vetharoy, Winston; Hu, Yi-Ting; Rai, Rajeev; Gomez-Castaneda, Eduardo; Cunha-Santos, Catarina; Burns, Siobhan O.; Morris, Emma C.; Booth, Claire; Turchiano, Giandomenico; Cavazza, Alessia; Thrasher, Adrian J.; Santilli, Giorgia
AAV capsid bioengineering in primary human retina models
SCIENTIFIC REPORTS
IF3.9
2023-12-117
OAAI
Westhaus, Adrian; Eamegdool, Steven S.; Fernando, Milan; Fuller-Carter, Paula; Brunet, Alicia A.; Miller, Annie L.; Rashwan, Rabab; Knight, Maddison; Daniszewski, Maciej; Lidgerwood, Grace E.; Pebay, Alice; Hewitt, Alex; Santilli, Giorgia; Thrasher, Adrian J.; Carvalho, Livia S.; Gonzalez-Cordero, Anai; Jamieson, Robyn V.; Lisowski, Leszek
Replication competent retrovirus testing (RCR) in the National Gene Vector Biorepository: No evidence of RCR in 1,595 post-treatment peripheral blood samples obtained from 60 clinical trials
MOLECULAR THERAPY
IF12
2023-03-016
OAAI
Cornetta, Kenneth; Yao, Jing; House, Kimberley; Duffy, Lisa; Adusumilli, Prasad S.; Beyer, Rachel; Booth, Claire; Brenner, Malcolm; Curran, Kevin; Grilley, Bambi; Heslop, Helen; Hinrichs, Christian S.; Kaplan, Rosandra N.; Kiem, Hans-Peter; Kochenderfer, James; Kohn, Donald B.; Mailankody, Sham; Norberg, Scott M.; O'Cearbhaill, Roisin E.; Pappas, Jennifer; Park, Jae; Ramos, Carlos; Ribas, Antonio; Riviere, Isabelle; Rosenberg, Steven A.; Sauter, Craig; Shah, Nirali N.; Slovin, Susan F.; Thrasher, Adrian; Williams, David A.; Lin, Tsai-Yu
Access to gene therapy for rare diseases when commercialization is not fit for purpose

当商业化不适合目的时，获得罕见疾病的基因治疗

NATURE MEDICINE
IF50
2023-02-1328
PRE
Fox, Thomas; Bueren, Juan; Candotti, Fabio; Fischer, Alain; Aiuti, Alessandro; Lankester, Arjan; AGORA Initiative, Michael; Albert, Michael; Bernardo, Maria Ester; Cavazzana, Marina; Ehl, Stephan; Neven, Benedicte; Prevot, Johan; Schambach, Axel; Sevilla, Julian; Thrasher, Adrian; Wynn, Rob; Booth, Claire
Severe hematopoietic stem cell inflammation compromises chronic granulomatous disease gene therapy
CELL REPORTS MEDICINE
IF10.6
2023-02-019
OAAI
Sobrino, Steicy; Magnani, Alessandra; Semeraro, Michaela; Martignetti, Loredana; Cortal, Akira; Denis, Adeline; Couzin, Chloe; Picard, Capucine; Bustamante, Jacinta; Magrin, Elisa; Joseph, Laure; Roudaut, Cecile; Gabrion, Aurelie; Soheili, Tayebeh; Cordier, Corinne; Lortholary, Olivier; Lefrere, Francois; Rieux-Laucat, Frederic; Casanova, Jean-Laurent; Bodard, Sylvain; Boddaert, Nathalie; Thrasher, Adrian J.; Touzot, Fabien; Taque, Sophie; Suarez, Felipe; Marcais, Ambroise; Guilloux, Agathe; Lagresle-Peyrou, Chantal; Galy, Anne; Rausell, Antonio; Blanche, Stephane; Cavazzana, Marina; Six, Emmanuelle
Partial human Janus kinase 1 deficiency predominantly impairs responses to interferon gamma and intracellular control of mycobacteria
FRONTIERS IN IMMUNOLOGY
IF5.9
2022-09-099
OAAI
Daza-Cajigal, Vanessa; Albuquerque, Adriana S.; Young, Dan F.; Ciancanelli, Michael J.; Moulding, Dale; Angulo, Ivan; Jeanne-Julien, Valentine; Rosain, Jeremie; Minskaia, Ekaterina; Casanova, Jean-Laurent; Boisson-Dupuis, Stephanie; Bustamante, Jacinta; Randall, Richard E.; McHugh, Timothy D.; Thrasher, Adrian J.; Burns, Siobhan O.

研究方向

暂未获取到该数据
M
Michael P. Blundell
H 指数:34论文数:94被引数:3,852
H
H. Bobby Gaspar
H 指数:47论文数:112被引数:9,317
S
Siobhan O. Burns
H 指数:53论文数:225被引数:1.1万
K
Kimberly Gilmour
H 指数:67论文数:293被引数:2.1万
C
Claire Booth
H 指数:41论文数:175被引数:6,031
M
Manfred Schmidt
H 指数:83论文数:488被引数:3.5万
C
CoryGiles
H 指数:44论文数:226被引数:1.0万
S
Steven J. Howe
H 指数:33论文数:113被引数:5,482
G
Giorgia Santilli
H 指数:29论文数:98被引数:3,354
W
Waseem Qasim
H 指数:55论文数:267被引数:1.0万