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Journal Articles

Long Term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed up to 7 Years Post-Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy and Exploratory Analysis of Predictors of Successful Treatment Outcomes in Phase 3 Trials

Blood (2022) 140 (Supplement 1): 5355–5357.

https://doi.org/10.1182/blood-2022-162400

Published: 2022

Journal Articles

Long-Term Patient-Reported Outcomes Following Treatment with betibeglogene autotemcel in Patients with Transfusion-Dependent β-Thalassemia

Franco Locatelli, Mark C. Walters, Janet L. Kwiatkowski, Marina Cavazzana, Suradej Hongeng, John B. Porter, Adrian J. Thrasher, Andreas E. Kulozik, Isabelle Thuret, John E.J. Rasko, Evangelia Yannaki, Martin G. Sauer, Shamshad Ali, Himal Thakar, Katiana Gruppioni, Alexis A. Thompson

Blood (2022) 140 (Supplement 1): 8200–8202.

https://doi.org/10.1182/blood-2022-163178

Published: 2022

Journal Articles

Long-Term Durable FVIII Expression with Improvements in Bleeding Rates Following AAV-Mediated FVIII Gene Transfer for Hemophilia A: Multiyear Follow-up on the Phase I/II Trial of SPK-8011

Blood (2022) 140 (Supplement 1): 1899–1901.

https://doi.org/10.1182/blood-2022-158903

Published: 2022

Journal Articles

Rapid Clearance of Vector Following AAV-Mediated FVIII Gene Transfer in the Phase I/II Trial of SPK-8011 in People with Hemophilia A

Huyen Tran, M. Elaine Eyster, Stacy E. Croteau, Margaret V. Ragni, Benjamin J. Samelson-Jones, Spencer Sullivan, John E.J. Rasko, Kristen Jaworski, Amy MacDougall, Savina Jaeger, Magdalena Hofer, Charlie Li, Federico Mingozzi, Tiffany Chang, Gallia Levy

Blood (2022) 140 (Supplement 1): 10662–10664.

https://doi.org/10.1182/blood-2022-159027

Published: 2022

Journal Articles

The Effects of Immunomodulation with Corticosteroids to Manage an AAV Capsid Immune response in the Phase I/II Study of SPK-8011

Matthew S. Evans, Witold B. Rybka, Stacy E. Croteau, Huyen Tran, John E.J. Rasko, Kristen Jaworski, Amy MacDougall, Savina Jaeger, Federico Mingozzi, Tiffany Chang, Gallia Levy

Blood (2022) 140 (Supplement 1): 10654–10655.

https://doi.org/10.1182/blood-2022-159017

Published: 2022

Journal Articles

Follow-up of More Than 5 Years in a Cohort of Patients with Hemophilia B Treated with Fidanacogene Elaparvovec Adeno-Associated Virus Gene Therapy

Ben J. Samelson-Jones, Spencer K. Sullivan, John E.J. Rasko, Adam Giermasz, Lindsey A. George, Jonathan M. Ducore, Jerome M. Teitel, Catherine E. McGuinn, Amanda O'Brien, Ian Winburn, Lynne M Smith, Amit Chhabra, Jeremy Rupon

Blood (2021) 138 (Supplement 1): 3975.

https://doi.org/10.1182/blood-2021-150541

Published: 2021

Journal Articles

Restoring Iron Homeostasis in Pts Who Achieved Transfusion Independence after Treatment with Betibeglogene Autotemcel Gene Therapy: Results from up to 7 Years of Follow-up

Alexis A. Thompson, Franco Locatelli, Evangelia Yannaki, Mark C. Walters, John B. Porter, Suradej Hongeng, Andreas E. Kulozik, Martin G. Sauer, Adrian J. Thrasher, Isabelle Thuret, Ashutosh Lal, Marina Cavazzana, John E.J. Rasko, Lin Du, Richard A. Colvin, Janet L. Kwiatkowski

Blood (2021) 138 (Supplement 1): 573.

https://doi.org/10.1182/blood-2021-148177

Published: 2021

Journal Articles

Long-Term Efficacy and Safety of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia: Results in Patients with up to 6 Years of Follow-up

Janet L. Kwiatkowski, MD MSCE, Mark C. Walters, MD, Suradej Hongeng, MD, Franco Locatelli, MD PhD, John E.J. Rasko, BSc, MBBS, PhD, Marina Cavazzana, MD PhD, Ying Chen, PhD, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD

Blood (2020) 136 (Supplement 1): 51–52.

https://doi.org/10.1182/blood-2020-135850

Published: 2020

Journal Articles

Efficacy and Safety of PD-1 Inhibitor Plus GVD Chemotherapy in Patients with Relapsed/Refractory Primary Mediastinal Large B-Cell Lymphoma (rrPMLBCL)

Qian Mei, PhD, Wenying Zhang, MD, Yang Liu, MD, Qingming Yang, MD, John E.J. Rasko, MBBS, PhD, Weidong Han, PhD MD

Blood (2019) 134 (Supplement_1): 4072.

https://doi.org/10.1182/blood-2019-121768

Published: 2019

Journal Articles

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

Janet L. Kwiatkowski, MD MSCE, Alexis A. Thompson, MD MPH, John E.J. Rasko, MBBS, PhD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, MD, Marina Cavazzana, MD PhD, P. Joy Ho, MBBS DPhil FRACP FRCPA, Manfred Schmidt, PhD, Morris Kletzel, MD MBA, Elliott P. Vichinsky, MD, Briana Deary, Ying Chen, PhD, Alexandria Petrusich, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 4628.

https://doi.org/10.1182/blood-2019-125807

Published: 2019

Journal Articles

Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year

Lindsey A. George, MD, Spencer K. Sullivan, MD, John E.J. Rasko, MBBS, PhD, Adam Giermasz, MD PhD, Benjamin J. Samelson-Jones, MD PhD, Jonathan M. Ducore, MD MPH, Jerome M. Teitel, MD FRCPC, Catherine E. McGuinn, MD, Alexa R. Runowski, MS, Fraser Wright, PhD, Xavier M. Anguela, PhD, Katherine A. High, MD, Denis Rybin, PhD, John E. Murphy, PhD, Jeremy Rupon, MD

Blood (2019) 134 (Supplement_1): 3347.

https://doi.org/10.1182/blood-2019-124091

Published: 2019

Journal Articles

A Phase I Trial of iPSC-Derived MSCs (CYP-001) in Steroid-Resistant Acute GvHD

Adrian Bloor, MA MB BChir PhD FCRP FRCPath, Amit Patel, MBBS, PhD MRCP(Lond), MRCS(Eng), MAcadMEd, FFICM, FRCPath, James E Griffin, BSc (Hons), MBChB (Hons), PhD, Maria H Gilleece, MD FRCPath, MB, BSc, FRCP, Rohini Radia, MD FRCPath, David T Yeung, PhD BSc, FRACP, FRCPA, MBBS, Igor Slukvin, MD PhD, Kilian Kelly, PhD, John E.J. Rasko, MBBS, PhD

Blood (2018) 132 (Supplement 1): 4562.

https://doi.org/10.1182/blood-2018-99-110432

Published: 2018

Journal Articles

Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia Following Completion of the Northstar HGB-204 Study

John E.J. Rasko, MBBS, PhD, Alexis A. Thompson, MD MPH, Janet L. Kwiatkowski, MD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, MD, Marina Cavazzana, MD PhD, P. Joy Ho, MBBS DPhil FRACP FRCPA, Manfred Schmidt, PhD, Morris Kletzel, MD MBA, Philippe Leboulch, MD, Elliott Vichinsky, MD, Briana Deary, Ying Chen, PhD, Mohammed Asmal, MD PhD, Mark C. Walters, MD

Blood (2018) 132 (Supplement 1): 167.

https://doi.org/10.1182/blood-2018-167

Published: 2018

Journal Articles

Mesenchymal Stem Cells as Suicide Gene Therapy Vehicles for Organ-Confined and Metastatic Prostate Cancer (PCa).

Rosetta Martiniello-Wilks, BSc (Hons), PhD, Stephen R. Larsen, MBBS(Hons), Stephane Flamant, BSc (Hons), PhD, Jessamy C. Tiffen, BSc (Hons), Charles G. Bailey, BSc (Hons), PhD, John E.J. Rasko, BSc(Med), MBBS, PhD

Blood (2007) 110 (11): 5148.

https://doi.org/10.1182/blood.V110.11.5148.5148

Published: 2007

Journal Articles

Optimizing Mobilization, Augmentation, and Gene Transfer of Multipotent Mesenchymal Stromal Cells (MSCs).

Stephen R. Larsen, MBBS, Keefe Chng, BVSc, Shangzhen Zhou, John Wright, PhD, Margaret Armstrong, Sally Thompson, BVSc, Annemarie Hennessy, MBBS PhD, Rosetta Martiniello-Wilks, BSc PhD, John Gibson, MBBS PhD, Douglas E. Joshua, MBBS PhD, John E.J. Rasko, MBBS PhD

Blood (2006) 108 (11): 2555.

https://doi.org/10.1182/blood.V108.11.2555.2555

Published: 2006

Journal Articles

AAV-2 Capsid-Specific CD8+ T Cells Limit the Duration of Gene Therapy in Humans and Cross-React with AAV-8 Capsid.

Federico Mingozzi, PhD, Marcela V. Maus, MD,PhD, Denise E. Sabatino, PhD, Daniel J. Hui, PhD, John E.J. Rasko, MBBS, PhD, Margaret V. Ragni, MD, Catherine Scott Manno, MD, Hildegund C.J. Ertl, MD, Katherine A. High, MD

Blood (2006) 108 (11): 455.

https://doi.org/10.1182/blood.V108.11.455.455

Published: 2006

Journal Articles

Addition of Pegylated Megakaryocyte Growth Development Factor (pegMGDF) to G-CSF Improves the Mobilization of Primitive Hemopoietic Cells.

Stephen R. Larsen, MBBS, Keefe Chng, BVSc, Margaret Armstrong, DipNSSC(Aph), Fiona Battah, BSc, PhD, Marcus Hayward, BSc, Louie Leung, BSc, Sally Thomson, BVSc, Annemarie Hennessy, MBBS, PhD, John Gibson, MBBS, PhD, Douglas E. Joshua, MBBS, PhD, John E.J. Rasko, MBBS, PhD

Blood (2005) 106 (11): 1967.

https://doi.org/10.1182/blood.V106.11.1967.1967

Published: 2005

Journal Articles

Improved Gene Transfer Into Baboon Marrow Repopulating Cells Using Recombinant Human Fibronectin Fragment CH-296 in Combination With Interleukin-6, Stem Cell Factor, FLT-3 Ligand, and Megakaryocyte Growth and Development Factor

Hans-Peter Kiem, Robert G. Andrews, Julia Morris, Laura Peterson, Scott Heyward, James M. Allen, John E.J. Rasko, Jennifer Potter, A. Dusty Miller

Blood (1998) 92 (6): 1878–1886.

https://doi.org/10.1182/blood.V92.6.1878

Published: 1998

Journal Articles

Randomized, Blinded, Placebo-Controlled Phase I Trial of Pegylated Recombinant Human Megakaryocyte Growth and Development Factor With Filgrastim After Dose-Intensive Chemotherapy in Patients With Advanced Cancer

Russell L. Basser, John E.J. Rasko, Kerrie Clarke, Jonathan Cebon, Michael D. Green, Andrew P. Grigg, John Zalcberg, Brian Cohen, Joan O'Byrne, Dora M. Menchaca, Richard M. Fox, C. Glenn Begley

Blood (1997) 89 (9): 3118–3128.

https://doi.org/10.1182/blood.V89.9.3118

Published: 1997

Journal Articles

Cytokine Receptor Expression on Hematopoietic Stem and Progenitor Cells

William J. McKinstry, Chung-Leung Li, John E.J. Rasko, Nicos A. Nicola, Gregory R. Johnson, Donald Metcalf

Blood (1997) 89 (1): 65–71.

https://doi.org/10.1182/blood.V89.1.65

Published: 1997

NARROW

Long Term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed up to 7 Years Post-Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy and Exploratory Analysis of Predictors of Successful Treatment Outcomes in Phase 3 Trials

Long-Term Patient-Reported Outcomes Following Treatment with betibeglogene autotemcel in Patients with Transfusion-Dependent β-Thalassemia

Long-Term Durable FVIII Expression with Improvements in Bleeding Rates Following AAV-Mediated FVIII Gene Transfer for Hemophilia A: Multiyear Follow-up on the Phase I/II Trial of SPK-8011

Rapid Clearance of Vector Following AAV-Mediated FVIII Gene Transfer in the Phase I/II Trial of SPK-8011 in People with Hemophilia A

The Effects of Immunomodulation with Corticosteroids to Manage an AAV Capsid Immune response in the Phase I/II Study of SPK-8011

Follow-up of More Than 5 Years in a Cohort of Patients with Hemophilia B Treated with Fidanacogene Elaparvovec Adeno-Associated Virus Gene Therapy

Restoring Iron Homeostasis in Pts Who Achieved Transfusion Independence after Treatment with Betibeglogene Autotemcel Gene Therapy: Results from up to 7 Years of Follow-up

Long-Term Efficacy and Safety of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia: Results in Patients with up to 6 Years of Follow-up

Efficacy and Safety of PD-1 Inhibitor Plus GVD Chemotherapy in Patients with Relapsed/Refractory Primary Mediastinal Large B-Cell Lymphoma (rrPMLBCL)

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year

A Phase I Trial of iPSC-Derived MSCs (CYP-001) in Steroid-Resistant Acute GvHD

Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia Following Completion of the Northstar HGB-204 Study

Mesenchymal Stem Cells as Suicide Gene Therapy Vehicles for Organ-Confined and Metastatic Prostate Cancer (PCa).

Optimizing Mobilization, Augmentation, and Gene Transfer of Multipotent Mesenchymal Stromal Cells (MSCs).

AAV-2 Capsid-Specific CD8+ T Cells Limit the Duration of Gene Therapy in Humans and Cross-React with AAV-8 Capsid.

Addition of Pegylated Megakaryocyte Growth Development Factor (pegMGDF) to G-CSF Improves the Mobilization of Primitive Hemopoietic Cells.

Improved Gene Transfer Into Baboon Marrow Repopulating Cells Using Recombinant Human Fibronectin Fragment CH-296 in Combination With Interleukin-6, Stem Cell Factor, FLT-3 Ligand, and Megakaryocyte Growth and Development Factor

Randomized, Blinded, Placebo-Controlled Phase I Trial of Pegylated Recombinant Human Megakaryocyte Growth and Development Factor With Filgrastim After Dose-Intensive Chemotherapy in Patients With Advanced Cancer

Cytokine Receptor Expression on Hematopoietic Stem and Progenitor Cells

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American Society of Hematology

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American Society of Hematology

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