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Journal Articles

Comparative Analysis of CRISPR-Cas9, lentiviral transduction, and base editing for sickle cell disease in a murine model

Henna Butt, Shruti Sathish, Evan London, Anh Le, Quan Li, Bjorg Gudmundsdottir, Duck-Yeon Lee, Emma V Burke, Bradley P Yates, David R Liu, Matthew M Hsieh, Alexis Leonard, William A Eaton, Naoya Uchida, Francis J. Pierciey, Gregory A Newby, John F. Tisdale, Selami Demirci

Blood Adv bloodadvances.2025017321.

https://doi.org/10.1182/bloodadvances.2025017321

Published: 2025

Journal Articles

Real-world data for tisagenlecleucel in patients with R/R B-ALL: subgroup analyses from the CIBMTR registry

Blood Adv (2025) 9 (20): 5249–5262.

https://doi.org/10.1182/bloodadvances.2025015881

Published: 2025

Includes: Supplemental data

Journal Articles

Informed Shared Decision-Making or Rigid Eligibility and Exclusion Criteria for Gene Therapy for Sickle Cell Disease?

Lakshmanan Krishnamurti, Ayobami Olafimihan, Lewis L Hsu

Blood Adv bloodadvances.2025017015.

https://doi.org/10.1182/bloodadvances.2025017015

Published: 2025

Journal Articles

Point-of-care fresh CAR T cells for pediatric or young adult BCP-ALL that is relapsed/refractory or in very-high-risk first relapse

Blood Adv (2025) 9 (19): 5091–5102.

https://doi.org/10.1182/bloodadvances.2025016181

Published: 2025

Includes: Supplemental data

Journal Articles

Point-of-care CAR T-cell therapy: toward broader access

Susana Rives

Blood Adv (2025) 9 (19): 5103–5104.

https://doi.org/10.1182/bloodadvances.2025017298

Published: 2025

Journal Articles

In situ gene editing of hematopoietic stem cells via AAV-delivered CRISPR guide RNAs

Alborz Karimzadeh, Rebekah Kim, Vivian Garcia, Michael Florea, Bryan L Peacker, Shio Kobayashi, Drake Watkins, Kathleen A. Messemer, Jing Zeng, Daniel E Bauer, Thomas Serwold, Amy J Wagers

Blood Adv bloodadvances.2025016775.

https://doi.org/10.1182/bloodadvances.2025016775

Published: 2025

Includes: Supplemental data

Journal Articles

Long-term outcomes of genome-edited “universal” CAR19 T cells for relapsed/refractory B-ALL at a single pediatric center

Daniela Guardo, Avijeet Kumar Mishra, Hebatalla Rashed, Kimberly Gilmour, Stuart Adams, Danielle Pinner, Martin Sauer, Ajay Vora, Paul Veys, Vesna Pavasovic, Kanchan Rao, Waseem Qasim

Blood Adv (2025) 9 (18): 4750–4754.

https://doi.org/10.1182/bloodadvances.2025016366

Published: 2025

Includes: Supplemental data

Journal Articles

Gene-edited CAR T cells: going the distance in B-ALL

John A. Ligon, Nirali N. Shah

Blood Adv (2025) 9 (18): 4755–4756.

https://doi.org/10.1182/bloodadvances.2025017508

Published: 2025

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 (2025) 9 (17): 4502–4512.

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

Published: 2025

Includes: Supplemental data

Journal Articles

Reality bites: allocating gene therapy for hemoglobinopathies

Venée N. Tubman, Titilope A. Fasipe

Blood Adv (2025) 9 (17): 4500–4501.

https://doi.org/10.1182/bloodadvances.2025016943

Published: 2025

Journal Articles

RAG1 lentiviral gene therapy restores T cell development of RAG1-SCID patient cells in artificial thymic organoids

Xiaolin Meng, Janine E Melsen, Rosalie van der Holst, Bas de Mooij, Sandra Vloemans, Marja van Eggermond, Dagmar Berghuis, Arjan C Lankester, Sander de Kivit, Karin Pike-Overzet, Anton W. Langerak, Frank JT Staal, Lisa M Ott de Bruin, Kirsten Canté-Barrett

Blood Adv bloodadvances.2025016970.

https://doi.org/10.1182/bloodadvances.2025016970

Published: 2025

Includes: Supplemental data

Journal Articles

To pursue gene therapy or not? Is it feasible after graft failure in allogeneic hematopoietic cell transplant recipients

Akshay Sharma, Tami John

Blood Adv (2025) 9 (15): 3845–3852.

https://doi.org/10.1182/bloodadvances.2024015413

Published: 2025

Journal Articles

Getting closer to hemophilia gene therapy for all?

Ulrike M. Reiss

Blood Adv (2025) 9 (14): 3629–3630.

https://doi.org/10.1182/bloodadvances.2025016642

Published: 2025

Journal Articles

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

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

Blood Adv (2025) 9 (14): 3543–3552.

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

Published: 2025

Includes: Supplemental data

Journal Articles

Long-term data needed for SCD gene therapy

Alexis Leonard

Blood Adv (2025) 9 (12): 3104–3105.

https://doi.org/10.1182/bloodadvances.2025016267

Published: 2025

Journal Articles

Gene therapy for hemophilia B: results from the phase 1/2 101HEMB01/02 studies

Steven Pipe, Allen Poma, Anita Rajasekhar, Tamara Everington, Serap Sankoh, Jack Allen, Jason Cataldo, Eric Crombez

Blood Adv (2025) 9 (12): 2980–2987.

https://doi.org/10.1182/bloodadvances.2024015184

Published: 2025

Journal Articles

Extracellular vesicles for CAR T-cell therapy immunomonitoring

Marion Klea Pinheiro, Benoît Vingert

Blood Adv (2025) 9 (12): 2920–2921.

https://doi.org/10.1182/bloodadvances.2025015988

Published: 2025

Journal Articles

CD19.CAR T-cell–derived extracellular vesicles express CAR and kill leukemic cells, contributing to antineoplastic therapy

Blood Adv (2025) 9 (12): 2907–2919.

https://doi.org/10.1182/bloodadvances.2024014860

Published: 2025

Includes: Supplemental data

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

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

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Comparative Analysis of CRISPR-Cas9, lentiviral transduction, and base editing for sickle cell disease in a murine model

Real-world data for tisagenlecleucel in patients with R/R B-ALL: subgroup analyses from the CIBMTR registry

Informed Shared Decision-Making or Rigid Eligibility and Exclusion Criteria for Gene Therapy for Sickle Cell Disease?

Point-of-care fresh CAR T cells for pediatric or young adult BCP-ALL that is relapsed/refractory or in very-high-risk first relapse

Point-of-care CAR T-cell therapy: toward broader access

In situ gene editing of hematopoietic stem cells via AAV-delivered CRISPR guide RNAs

Long-term outcomes of genome-edited “universal” CAR19 T cells for relapsed/refractory B-ALL at a single pediatric center

Gene-edited CAR T cells: going the distance in B-ALL

An ethical allocation scheme for scarce gene therapies in sickle cell disease and transfusion-dependent β-thalassemia

Reality bites: allocating gene therapy for hemoglobinopathies

RAG1 lentiviral gene therapy restores T cell development of RAG1-SCID patient cells in artificial thymic organoids

To pursue gene therapy or not? Is it feasible after graft failure in allogeneic hematopoietic cell transplant recipients

Getting closer to hemophilia gene therapy for all?

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

Long-term data needed for SCD gene therapy

Gene therapy for hemophilia B: results from the phase 1/2 101HEMB01/02 studies

Extracellular vesicles for CAR T-cell therapy immunomonitoring

CD19.CAR T-cell–derived extracellular vesicles express CAR and kill leukemic cells, contributing to antineoplastic therapy

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

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

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