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Adrian J. Thrasher, MBBS, PhD FMedSci
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Journal Articles

Response of Patients with Transfusion-Dependent β-Thalassemia (TDT) to Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-Thalassemia) Gene Therapy Based on HBB Genotype and Disease Genetic Modifiers

Mark C. Walters, MD, David H.K. Chui, MD FRCPC, John J Farrell, PhD, Ashutosh Lal, MD, Franco Locatelli, MD PhD, Janet L. Kwiatkowski, MD MSCE, John B. Porter, MA, MD, FRCP, FRCPath, Martin G. Sauer, MD, Isabelle Thuret, MD, Suradej Hongeng, MD, Andreas E. Kulozik, MD PhD, Adrian J. Thrasher, MBBS, PhD FMedSci, Evangelia Yannaki, MD, Julia Yang, Dustin Whitney, PhD, Alexandria Petrusich, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD
Journal: Blood
Blood (2020) 136 (Supplement 1): 1–3.
https://doi.org/10.1182/blood-2020-137642
Published: 2020
Journal Articles

Ex-Vivo Autologous Stem Cell Gene Therapy Clinical Trial for Mucopolysaccharidosis Type IIIA: Trial in Progress - NCT04201405

Jane L Kinsella, MBChB, Robert F Wynn, MRCP,MDFRCPath, Brian Bigger, PhD, Adrian J. Thrasher, MBBS, PhD FMedSci, Claire Booth, MBBS, PhD MSc, Karen Buckland, PhD, Natalia Izotova, Stewart Rust, B.Sc. (Hons), D.Clin.Psych, Pg.Dip., C.Psychol., AfBPS., Daniel Weisberg, BSc, PhD PGCert, ClinPsyD, Heather J Church, PhD, Kathryn L Brammeier, Karen L Tylee, Ceri Jones, BSc, Helena Lee, Laura Ford, Atusa Sadegholnejat, Stuart Ellison, Simon A Jones, MBChB, BSc, MRCPCH
Journal: Blood
Blood (2020) 136 (Supplement 1): 15–16.
https://doi.org/10.1182/blood-2020-141762
Published: 2020
Journal Articles

Editing the LRF Repressor Binding Site in the γ-Globin Promoters Induces Therapeutically Relevant Fetal Hemoglobin Levels for the Treatment of β-Hemoglobinopathies

Giacomo Frati, PhD, Panagiotis Antoniou, MSc, Giulia Hardouin, Bochra Mlaya, MSc, Leslie Weber, Tristan Felix, Antonio Casini, Marion Rosello, Anne Chalumeau, Mario Amendola, Adrian J. Thrasher, MBBS, PhD FMedSci, Giandomenico Turchiano, PhD, Filippo Del Bene, Wassim El Nemer, PhD, Anna Cereseto, Jean-Paul Concordet, Carine Giovannangeli, Marina Cavazzana, MD PhD, Annarita Miccio, PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 33.
https://doi.org/10.1182/blood-2020-139107
Published: 2020
Journal Articles

Favorable Outcomes in Pediatric Patients in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia

Alexis A. Thompson, MD, Janet L. Kwiatkowski, MD MSCE, John B. Porter, MA, MD, FRCP, FRCPath, Suradej Hongeng, MD, Evangelia Yannaki, MD, Andreas E. Kulozik, MD PhD, Martin G. Sauer, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, Isabelle Thuret, MD, Ashutosh Lal, MD, Ruiting Guo, MS, Weijian Liu, PhD, Richard A. Colvin, MD PhD, Mark C. Walters, MD, Franco Locatelli, MD PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 52–54.
https://doi.org/10.1182/blood-2020-135857
Published: 2020
Journal Articles

Bipotent Lymphoid Progenitors Independently Maintain Long-Term Genetically Engineered T and NK Cell Production in Humans

Natalia Izotova, Christine Rivat, Cristina Baricordi, PhD, Elena Blanco-Alvarez, Danilo Pellin, Eleanor Watt, Soragia Athina Gkazi, PhD, Stuart Adams, PhD, Kimberly Gilmour, Jinhua Xu Bayford, Claire Booth, MBBS, PhD MSc, Bobby H Gaspar, MBBS, PhD BS, Adrian J. Thrasher, MBBS, PhD FMedSci, Luca Biasco, PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 50.
https://doi.org/10.1182/blood-2020-137001
Published: 2020
Journal Articles

Northstar-2: Updated Safety and Efficacy Analysis of Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Non-β0/β0 Genotypes

Alexis A. Thompson, MD MPH, Mark C. Walters, MD, Janet L. Kwiatkowski, MD MSCE, Suradej Hongeng, MD, John B. Porter, MA MDFRCP FRCPath, Martin G. Sauer, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, Isabelle Thuret, MD, Heidi Elliot, Ge Tao, PhD, Richard A. Colvin, MD PhD, Franco Locatelli, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3543.
https://doi.org/10.1182/blood-2019-126046
Published: 2019
Journal Articles

Clonal Dynamics of Early Responder and Long-Term Surviving CAR-T Cells in Humans

Christine Rivat, Natalia Izotova, Rachel Richardson, MSc, Danilo Pellin, Rachael Hough, Robert Wynn, MD, Kim Champion, Andre Lopes, Sara Ghorashian, FRCPath, PhD, Martin Pule, Adrian J. Thrasher, MBBS, PhD FMedSci, Luca Biasco, PhD, Persis J Amrolia, FRCP, FRCPath, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 52.
https://doi.org/10.1182/blood-2019-125916
Published: 2019
Journal Articles

Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded Cohort

Donald B. Kohn, MD, Kit L. Shaw, PhD, Elizabeth Garabedian, RN, MS, Denise Ann Carbonaro-Sarracino, PhD, Theodore B. Moore, MD, Satiro N. De Oliveira, MD, Gay M. Crooks, MD, John Tse, Sally Shupien, Dayna Terrazas, Alejandra Davila, Amalia Icreverzi, PhD, Allen Yu, Krista M. Chun, Christian E. Casas, Provaboti Barman, PhD, Maritess Coronel, Beatriz Campo Fernandez, PhD, Ruixue Zhang, Roger P. Hollis, PhD, Chilenwa Uzowuru, Hilory Ricketts, Jinhua Xu Bayford, Valentina Trevisan, Serena Arduini, PhD, Frances Lynn, Mahesh Kudari, MBBS, MA, Andrea Spezzi, MD MBBS, Lilith Reeves, MS, MT(ASCP), Kenneth Cornetta, Robert A. Sokolic, MD, Roberta Parrott, BS, Rebecca Buckley, MD, Claire Booth, Fabio Candotti, MD, Harry L. Malech, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, H. Bobby Gaspar, MBBS, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3345.
https://doi.org/10.1182/blood-2019-123432
Published: 2019