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Journal Articles

Protection of CD33-modified hematopoietic stem cell progeny from CD33-directed CAR T cells in rhesus macaques

Nicholas E. Petty, Stefan Radtke, Greta Kanestrom, Emily Fields, Olivier Humbert, Salvatore Fiorenza, Mallory J. Llewellyn, George S. Laszlo, Justin Thomas, Zach Burger, Kyle Swing, Haiying Zhu, Keith R. Jerome, Cameron J. Turtle, Roland B. Walter, Hans-Peter Kiem

Blood Adv (2025) 9 (10): 2367–2378.

https://doi.org/10.1182/bloodadvances.2024015016

Published: 2025

Includes: Supplemental data

Journal Articles

An Ethical Allocation Scheme for Scarce Gene Therapies in Sickle Cell Disease and Transfusion-Dependent β-Thalassemia

Amar H. Kelkar, Maureen O. Achebe, Andrew Hantel

Blood Adv bloodadvances.2025016053.

https://doi.org/10.1182/bloodadvances.2025016053

Published: 2025

Includes: Supplemental data

Journal Articles

Completion of Phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years

Annette von Drygalski, M.D., Esteban Gomez, Adam Giermasz, Giancarlo Castaman, Nigel S. Key, Susan U. Lattimore, Frank W.G. Leebeek, Wolfgang A. Miesbach, Michael Recht, Paul E. Monahan, M.D., Sandra Le Quellec, M.D., Ph.D, Steven W. Pipe, M.D.

Blood Adv bloodadvances.2024015291.

https://doi.org/10.1182/bloodadvances.2024015291

Published: 2025

Includes: Supplemental data

Journal Articles

Nivolumab to restore T-cell fitness in CAR-T refractory multiple myeloma

Johannes M. Waldschmidt, Noori Sotudeh, Sankalp Arora, Tushara Vijaykumar, Praveen Anand, Hannah Stuart, Julia Frede, Tim Campbell, Shari M. Kaiser, Xirong Zheng, Nikhil C. Munshi, Kenneth C. Anderson, Hermann Einsele, Andrew J. Yee, Birgit Knoechel, Jens G. Lohr, Noopur S. Raje

Blood Adv (2025) 9 (5): 1132–1136.

https://doi.org/10.1182/bloodadvances.2024015285

Published: 2025

Includes: Supplemental data

Journal Articles

The transplant debate: defining innovation for sickle cell

Cécile Karsenty, Titilope Fasipe, Alexander I. Ngwube

Blood Adv (2025) 9 (4): 933–934.

https://doi.org/10.1182/bloodadvances.2024015400

Published: 2025

Journal Articles

Is allogeneic transplantation for sickle cell disease still relevant in the era of gene therapy?

Richard J. Jones, Adetola A. Kassim, Robert A. Brodsky, Michael R. DeBaun

Blood Adv (2025) 9 (4): 877–883.

https://doi.org/10.1182/bloodadvances.2024013693

Published: 2025

Journal Articles

Nonclinical evaluation of HBG1/2 and BCL11A as genome-editing targets for the treatment of β-hemoglobinopathies

Tusneem Janoudi, Mrunali Jagdale, Meng Wu, Sanjana Gorlla, Peisheng Zhang, Ying Shao, Li Li, Sheryl R. Bowley, Eugenio Marco, Kai-Hsin Chang

Blood Adv (2025) 9 (4): 808–813.

https://doi.org/10.1182/bloodadvances.2024014040

Published: 2025

Includes: Supplemental data

Journal Articles

Induction of factor VIII tolerance by hemophilia gene transfer to eradicate factor VIII inhibitors

Guy Young

Blood Adv (2025) 9 (2): 265–269.

https://doi.org/10.1182/bloodadvances.2024013000

Published: 2025

Journal Articles

Gene therapy in transfusion-dependent non-β 0 /β 0 genotype β-thalassemia: first real-world experience of beti-cel

Adil Mirza, Mona-Lisa Ritsert, Gloria Tao, Himal Thakar, Stephan Lobitz, Sabine Heine, Leila Koscher, Matthias Dürken, Anita Schmitt, Michael Schmitt, Petra Pavel, Sascha Laier, Donate Jakoby, Johann Greil, Joachim Kunz, Andreas Kulozik

Blood Adv (2024) 9 (1): 29–38.

https://doi.org/10.1182/bloodadvances.2024014104

Published: 2024

Includes: Supplemental data

Journal Articles

Gene therapy for hemophilia

Peter J. Lenting, Sylvia Fong

Blood Adv (2024) 8 (23): 6081.

https://doi.org/10.1182/bloodadvances.2024013864

Published: 2024

Journal Articles

Adeno-associated virus–based gene therapy for hemophilia A and B: a systematic review and meta-analysis

Saarang R. Deshpande, Keerthy Joseph, Jiayi Tong, Yong Chen, Allyson Pishko, Adam Cuker

Blood Adv (2024) 8 (23): 5957–5974.

https://doi.org/10.1182/bloodadvances.2024014111

Published: 2024

Includes: Supplemental data

Journal Articles

UM171 enhances fitness and engraftment of gene-modified hematopoietic stem cells from patients with sickle cell disease

Boya Liu, Denise Klatt, Yu Zhou, John P. Manis, Guy Sauvageau, Danilo Pellin, Christian Brendel, David A. Williams

Blood Adv (2024) 8 (22): 5885–5895.

https://doi.org/10.1182/bloodadvances.2024013932

Published: 2024

Includes: Supplemental data

Journal Articles

Liver-related aspects of valoctocogene roxaparvovec gene therapy for hemophilia A: expert guidance for clinical practice

Vincenzo La Mura, Vincenzo Cardinale, Raimondo De Cristofaro, Adriano De Santis, Giovanni Di Minno, Luca Fabris, Fabio Marra, Filomena Morisco, Flora Peyvandi, Maurizio Pompili, Cristina Santoro, Ezio Zanon, Giancarlo Castaman

Blood Adv (2024) 8 (22): 5725–5734.

https://doi.org/10.1182/bloodadvances.2024013750

Published: 2024

Journal Articles

Listening to patients and parents with sickle cell disease: the totality of gene therapy risks may outweigh the perceived benefits

Liza-Marie Johnson, Akshay Sharma, Yvonne Carroll, Dominique Goodson, Belinda N. Mandrell, Jami Gattuso, Amanda Young, Jacklyn Boggs, Benjamin S. Wilfond, Yoram Unguru

Blood Adv (2024) 8 (21): 5723–5724.

https://doi.org/10.1182/bloodadvances.2024012963

Published: 2024

Journal Articles

The critical role of symptom experience on risk tolerance for gene therapy

Juan Marcos Gonzalez Sepulveda, Shelby D. Reed, Marilyn Telen

Blood Adv (2024) 8 (21): 5721–5722.

https://doi.org/10.1182/bloodadvances.2024013811

Published: 2024

Journal Articles

Optimizing liver health before and after gene therapy for hemophilia A

Margaret V. Ragni, Henry Mead, Ype P. de Jong, Radoslaw Kaczmarek, Andrew D. Leavitt, Brian Long, Diane J. Nugent, Denise E. Sabatino, Sylvia Fong, Annette von Drygalski, Christopher E. Walsh, Bruce A. Luxon

Blood Adv (2024) 8 (19): 5203–5212.

https://doi.org/10.1182/bloodadvances.2024013059

Published: 2024

Journal Articles

Roctavian gene therapy for hemophilia A

Benjamin J. Samelson-Jones, Juliana C. Small, Lindsey A. George

Blood Adv (2024) 8 (19): 5179–5189.

https://doi.org/10.1182/bloodadvances.2023011847

Published: 2024

Includes: Supplemental data

Journal Articles

Adeno-associated virus and hepatocytes: frenemies?

Glenn F. Pierce

Blood Adv (2024) 8 (19): 5200–5202.

https://doi.org/10.1182/bloodadvances.2024013845

Published: 2024

Journal Articles

Utilizing epigenetic regulators to improve HSC-based lentiviral gene therapy

Parisa Tajer, Emin Onur Karakaslar, Kirsten Canté-Barrett, Brigitta A. E. Naber, Sandra A. Vloemans, Marja C. J. A. van Eggermond, Marie-Louise van der Hoorn, Erik van den Akker, Karin Pike-Overzet, Frank J. T. Staal

Blood Adv (2024) 8 (18): 4936–4947.

https://doi.org/10.1182/bloodadvances.2024013047

Published: 2024

Includes: Supplemental data

Journal Articles

A budget impact analysis of gene therapy for sickle cell disease: an updated analysis

Patrick C. DeMartino, Meredith B. Haag, Aaron B. Caughey, Joshua A. Roth

Blood Adv (2024) 8 (17): 4658–4661.

https://doi.org/10.1182/bloodadvances.2024013093

Published: 2024

Includes: Supplemental data

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Protection of CD33-modified hematopoietic stem cell progeny from CD33-directed CAR T cells in rhesus macaques

An Ethical Allocation Scheme for Scarce Gene Therapies in Sickle Cell Disease and Transfusion-Dependent β-Thalassemia

Completion of Phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years

Nivolumab to restore T-cell fitness in CAR-T refractory multiple myeloma

The transplant debate: defining innovation for sickle cell

Is allogeneic transplantation for sickle cell disease still relevant in the era of gene therapy?

Nonclinical evaluation of HBG1/2 and BCL11A as genome-editing targets for the treatment of β-hemoglobinopathies

Induction of factor VIII tolerance by hemophilia gene transfer to eradicate factor VIII inhibitors

Gene therapy in transfusion-dependent non-β 0 /β 0 genotype β-thalassemia: first real-world experience of beti-cel

Gene therapy for hemophilia

Adeno-associated virus–based gene therapy for hemophilia A and B: a systematic review and meta-analysis

UM171 enhances fitness and engraftment of gene-modified hematopoietic stem cells from patients with sickle cell disease

Liver-related aspects of valoctocogene roxaparvovec gene therapy for hemophilia A: expert guidance for clinical practice

Listening to patients and parents with sickle cell disease: the totality of gene therapy risks may outweigh the perceived benefits

The critical role of symptom experience on risk tolerance for gene therapy

Optimizing liver health before and after gene therapy for hemophilia A

Roctavian gene therapy for hemophilia A

Adeno-associated virus and hepatocytes: frenemies?

Utilizing epigenetic regulators to improve HSC-based lentiviral gene therapy

A budget impact analysis of gene therapy for sickle cell disease: an updated analysis

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