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Journal Articles

Global Seroprevalence of Neutralizing Antibodies Against Adeno-Associated Virus (AAV) Serotypes of Relevance to Gene Therapy

Professor John Rasko, George Bashirians, Christos Petropoulos, Terri Wrin, Yuvika Paliwal, Suryanarayan Somanathan, Michelle Casey, Candida da Fonseca Pereira, Ian Winburn, Amit Chhabra

Blood (2022) 140 (Supplement 1): 10668–10670.

https://doi.org/10.1182/blood-2022-158305

Published: 2022

Journal Articles

Clinical Outcomes up to 3 Years Following Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar Hgb-204 Study

Janet L. Kwiatkowski, MD, Alexis A Thompson, MD MPH, John Rasko, MBBS, PhD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, Marina Cavazzana, MD PhD, Phoebe Joy Ho, Christof von Kalle, MD, Morris Kletzel, MD MBA, Philippe Leboulch, MD, Elliott Vichinsky, MD, Briana Deary, Mohammed Asmal, MD PhD, Mark C. Walters, MD

Blood (2017) 130 (Supplement 1): 360.

https://doi.org/10.1182/blood.V130.Suppl_1.360.360

Published: 2017

Journal Articles

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Factor IX with Minimal Immune Response

Daniel J Hui, PhD BS, Yun Liu, PhD, Ruchi Patel, Yifeng Chen, MD, Lindsey A. George, MD, Spencer K. Sullivan, MD, Adam Giermasz, MD, John Rasko, MBBS, PhD, Ben J. Samelson-Jones, MD PhD, Jonathan M. Ducore, MD, Sue Dasen, MS, Marcus E. Carr, MD PhD, Xavier M. Anguela, PhD, Katherine A. High, MD

Blood (2017) 130 (Supplement 1): 2056.

https://doi.org/10.1182/blood.V130.Suppl_1.2056.2056

Published: 2017

Journal Articles

Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia: Update from the Northstar Hgb-204 Phase 1/2 Clinical Study

Alexis A. Thompson, MD MPH, Janet Kwiatkowski, MD, John Rasko, MBBS, PhD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, MD, Marina Cavazzana, MD PhD, P. Joy Ho, Christof von Kalle, MD, Morris Kletzel, MD FAAP, MBA, Philippe Leboulch, MD, Elliott Vichinsky, MD, Alexandria Petrusich, Mohammed Asmal, MD PhD, Mark C. Walters, MD

Blood (2016) 128 (22): 1175.

https://doi.org/10.1182/blood.V128.22.1175.1175

Published: 2016

Journal Articles

Update of Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex-Vivo with a Lentiviral Beta AT87Q-Globin Vector (LentiGlobin BB305 Drug Product)

Mark C. Walters, MD, John Rasko, MBBS, PhD, Suradej Hongeng, MD, Janet Kwiatkowski, MD, Gary J Schiller, MD, Morris Kletzel, MD, P. Joy Ho, MBBS, DPhil, Elliott Vichinsky, MD, Christof von Kalle, MD, Marina Cavazzana, MD PhD, Philippe Leboulch, MD, Alexandria Petrusich, Sandeep Soni, MD, Alexis A. Thompson, MD

Blood (2015) 126 (23): 201.

https://doi.org/10.1182/blood.V126.23.201.201

Published: 2015

Journal Articles

A Dynamic Intron Retention Program in the Mammalian Megakaryocyte and Erythrocyte Lineages

Christopher R Edwards, PhD, Rob Middleton, Xiuli An, MD PhD, Tejaswini Mishra, PhD, Narla Mohandas, DSc, Ross C. Hardison, PhD, John Rasko, MBBS, PhD, Gerd A. Blobel, MD PhD

Blood (2015) 126 (23): 2380.

https://doi.org/10.1182/blood.V126.23.2380.2380

Published: 2015

Journal Articles

MicroRNA Dysregulation in Newly Diagnosed Chronic Myeloid Leukaemia Patients

Jane E. A. Gordon, B Med, Charles G Bailey, PhD, John Rasko, MBBS, PhD, William Ritchie, PhD, Dale B. Watkins, B.Sc. (Hons), Deborah L. White, PhD, FFSc(RCPA), Tamara Leclercq, PhD, Timothy P Hughes, MD, MBBS

Blood (2013) 122 (21): 4985.

https://doi.org/10.1182/blood.V122.21.4985.4985

Published: 2013

Journal Articles

Intron Retention Coupled with Nonsense-Mediated Decay Determines Protein Expression and Nuclear Morphology in Granulopoiesis

John Rasko, MBBS, PhD, Justin Wong, PhD, William Ritchie, PhD, Ebner Olivia, BSc, Selbach Matthias, PhD, Jason WH Wong, PhD, Yizhou Huang, BSc, Dadi Gao, BSc, Maria Gonzalez, MSc, Kinsha Baidya, BSc, Annora Theong, BSc, Teh-Liane Khoo, MBBS, Charles G Bailey, PhD, Jeff Holst, PhD

Blood (2012) 120 (21): 112.

https://doi.org/10.1182/blood.V120.21.112.112

Published: 2012

Journal Articles

Human Immune Responses to AAV-2 Capsid May Limit Duration of Expression in Liver-Directed Gene Transfer in Humans with Hemophilia B.

Katherine High, MD, Michael Tigges, PhD, Catherine Manno, MD, Denise Sabatino, PhD, Valder Arruda, MD, Roland Herzog, PhD, Pradip Rustagi, MD, John Rasko, MD, Jurg Sommer, PhD, Kristen Jaworski, RN, Margaret Ragni, MD, Bertil Glader, MD, Ruth Lessard, Alvin Luk, PhD, Linda Couto, PhD, Haiyan Jiang, PhD, Glenn Pierce, MD, Mark Kay, MD

Blood (2004) 104 (11): 413.

https://doi.org/10.1182/blood.V104.11.413.413

Published: 2004

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Global Seroprevalence of Neutralizing Antibodies Against Adeno-Associated Virus (AAV) Serotypes of Relevance to Gene Therapy

Clinical Outcomes up to 3 Years Following Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar Hgb-204 Study

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Factor IX with Minimal Immune Response

Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia: Update from the Northstar Hgb-204 Phase 1/2 Clinical Study

Update of Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex-Vivo with a Lentiviral Beta AT87Q-Globin Vector (LentiGlobin BB305 Drug Product)

A Dynamic Intron Retention Program in the Mammalian Megakaryocyte and Erythrocyte Lineages

MicroRNA Dysregulation in Newly Diagnosed Chronic Myeloid Leukaemia Patients

Intron Retention Coupled with Nonsense-Mediated Decay Determines Protein Expression and Nuclear Morphology in Granulopoiesis

Human Immune Responses to AAV-2 Capsid May Limit Duration of Expression in Liver-Directed Gene Transfer in Humans with Hemophilia B.

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American Society of Hematology

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