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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 (2025) 9 (1): 29–38.

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

Published: 2025

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

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 bloodadvances.2024014040.

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

Published: 2024

Includes: Supplemental data

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

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

Guy Young

Blood Adv bloodadvances.2024013000.

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

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

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

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

Journal Articles

Blood biodistribution and vector shedding of valoctocogene roxaparvovec in people with severe hemophilia A

Suresh Agarwal, Krystal Sandza, Kristin Obrochta Moss, Monica Vora, Alisa Bowen, Brenna Bunch, Jennifer Holcomb, Tara M. Robinson, Kala Jayaram, Chris B. Russell, Stephen Zoog, Christian Vettermann, Joshua Henshaw

Blood Adv (2024) 8 (17): 4606–4615.

https://doi.org/10.1182/bloodadvances.2024013150

Published: 2024

Journal Articles

A simplified G-CSF–free procedure allows for in vivo HSC gene therapy of sickle cell disease in a mouse model

Chang Li, Anna K. Anderson, Peter Ruminski, Michael Rettig, Darja Karpova, Hans-Peter Kiem, John F. DiPersio, André Lieber

Blood Adv (2024) 8 (15): 4089–4101.

https://doi.org/10.1182/bloodadvances.2024012757

Published: 2024

Includes: Supplemental data

Journal Articles

Blockage of BCL-XL overcomes venetoclax resistance across BCL2 + lymphoid malignancies irrespective of BIM status

Alexandra Dolnikova, Dmitry Kazantsev, Magdalena Klanova, Eva Pokorna, Dana Sovilj, Cristina Daniela Kelemen, Liliana Tuskova, Eva Hoferkova, Marek Mraz, Karel Helman, Nikola Curik, Katerina Machova Polakova, Ladislav Andera, Marek Trneny, Pavel Klener

Blood Adv (2024) 8 (13): 3532–3543.

https://doi.org/10.1182/bloodadvances.2024012906

Published: 2024

Includes: Supplemental data

Journal Articles

HLA reduction of human T cells facilitates generation of immunologically multicompatible cellular products

Pascal M. Winterhalter, Linda Warmuth, Philipp Hilgendorf, Julius M. Schütz, Sarah Dötsch, Torsten Tonn, Luka Cicin-Sain, Dirk H. Busch, Kilian Schober

Blood Adv (2024) 8 (13): 3416–3426.

https://doi.org/10.1182/bloodadvances.2023011496

Published: 2024

Includes: Supplemental data

Journal Articles

In vivo measurement of RBC survival in patients with sickle cell disease before or after hematopoietic stem cell transplantation

Blood Adv (2024) 8 (7): 1806–1816.

https://doi.org/10.1182/bloodadvances.2023011397

Published: 2024

Includes: Supplemental data

Journal Articles

Hemophilia B and gene therapy: a new chapter with etranacogene dezaparvovec

Xavier M. Anguela, Katherine A. High

Blood Adv (2024) 8 (7): 1796–1803.

https://doi.org/10.1182/bloodadvances.2023010511

Published: 2024

NARROW

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

Gene therapy for hemophilia

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

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

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

Roctavian gene therapy for hemophilia A

Adeno-associated virus and hepatocytes: frenemies?

Optimizing liver health before and after gene therapy for hemophilia A

Utilizing epigenetic regulators to improve HSC-based lentiviral gene therapy

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

Blood biodistribution and vector shedding of valoctocogene roxaparvovec in people with severe hemophilia A

A simplified G-CSF–free procedure allows for in vivo HSC gene therapy of sickle cell disease in a mouse model

Blockage of BCL-XL overcomes venetoclax resistance across BCL2 + lymphoid malignancies irrespective of BIM status

HLA reduction of human T cells facilitates generation of immunologically multicompatible cellular products

In vivo measurement of RBC survival in patients with sickle cell disease before or after hematopoietic stem cell transplantation

Hemophilia B and gene therapy: a new chapter with etranacogene dezaparvovec

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American Society of Hematology

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ASH Publications

American Society of Hematology

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