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Journal Articles

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

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

Blood Adv bloodadvances.2024013150.

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

Published: 2024

Journal Articles

Roctavian Gene Therapy for Hemophilia A

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

Blood Adv bloodadvances.2023011847.

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

Published: 2024

Includes: Supplemental data

Journal Articles

Response to: 'In Response to, "Preferences for potential benefits and risks for gene therapy..."'

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

Blood Adv bloodadvances.2024013811.

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

Published: 2024

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

Utilizing Epigenetic Regulators to improve HSC-based lentiviral gene therapy

Parisa Tajer, Emin Onur Karakaslar, Kirsten Canté-Barrett, Brigitta Naber, Sandra Vloemans, Marja van Eggermond, Marie-Louise van der Hoorn, Erik B van den Akker, Karin Pike-Overzet, Frank JT Staal

Blood Adv bloodadvances.2024013047.

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

Published: 2024

Includes: Supplemental data

Journal Articles

In Response to, "Preferences for potential benefits and risks for gene therapy in the treatment of sickle cell disease"

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

Blood Adv bloodadvances.2024012963.

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

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 Nugent, Denise E. Sabatino, Sylvia Fong, Annette von Drygalski, M.D., Christopher e Walsh, Bruce Luxon

Blood Adv bloodadvances.2024013059.

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

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 Kate Anderson, Peter G Ruminski, Michael P Rettig, Darja Karpova, Hans-Peter Kiem, John F. DiPersio, André Lieber

Blood Adv bloodadvances.2024012757.

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

Published: 2024

Includes: Supplemental data

Journal Articles

Mending RAG2: gene editing for treatment of RAG2 deficiency

Elizabeth D. Hicks, Michael D. Keller

Blood Adv (2024) 8 (7): 1817–1819.

https://doi.org/10.1182/bloodadvances.2023012079

Published: 2024

Journal Articles

Long live the red blood cell: biotin tagging in SCD

Sarah McCuskee, Susanna Curtis

Blood Adv (2024) 8 (7): 1804–1805.

https://doi.org/10.1182/bloodadvances.2023012245

Published: 2024

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

Journal Articles

Genetically corrected RAG2 -SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency

Blood Adv (2024) 8 (7): 1820–1833.

https://doi.org/10.1182/bloodadvances.2023011766

Published: 2024

Includes: Supplemental data

Journal Articles

Clinical features of neurotoxicity after CD19 CAR T-cell therapy in mantle cell lymphoma

Esther H. Nie, Yi-Jiun Su, John H. Baird, Neha Agarwal, Sushma Bharadwaj, Wen-Kai Weng, Melody Smith, Saurabh Dahiya, May H. Han, Jeffrey E. Dunn, Lucas B. Kipp, David B. Miklos, Brian J. Scott, Matthew J. Frank

Blood Adv (2024) 8 (6): 1474–1486.

https://doi.org/10.1182/bloodadvances.2023011896

Published: 2024

Includes: Supplemental data

Journal Articles

CRISPR/Cas9 deletion of MIR155HG in human T cells reduces incidence and severity of acute GVHD in a xenogeneic model

Lotus Neidemire-Colley, Shrijan Khanal, Kara M. Braunreiter, Yandi Gao, Rathan Kumar, Katiri J. Snyder, Margot A. Weber, Simran Surana, Olimjon Toirov, Malith Karunasiri, Molly E. Duszynski, Mengna Chi, Punam Malik, Sonu Kalyan, Wing K. Chan, Meisam Naeimi Kararoudi, Hannah K. Choe, Ramiro Garzon, Parvathi Ranganathan

Blood Adv (2024) 8 (4): 947–958.

https://doi.org/10.1182/bloodadvances.2023010570

Published: 2024

Includes: Supplemental data

Journal Articles

Choosing the right mouse model: comparison of humanized NSG and NBSGW mice for in vivo HSC gene therapy

Seunga Choo, Carl B. Wolf, Heather M. Mack, Mitchell J. Egan, Hans-Peter Kiem, Stefan Radtke

Blood Adv (2024) 8 (4): 916–926.

https://doi.org/10.1182/bloodadvances.2023011371

Published: 2024

Includes: Supplemental data

Journal Articles

Answering the “Doctor, can CAR-T therapy cause cancer?” question in clinic

Rahul Banerjee, Christina Poh, Alexandre V. Hirayama, Jordan Gauthier, Ryan D. Cassaday, Mazyar Shadman, Andrew J. Cowan, Brian G. Till, Damian J. Green, Hans-Peter Kiem, Ajay K. Gopal, David G. Maloney

Blood Adv (2024) 8 (4): 895–898.

https://doi.org/10.1182/bloodadvances.2023012336

Published: 2024

Journal Articles

Cost-effectiveness of second-line lisocabtagene maraleucel in relapsed or refractory diffuse large B-cell lymphoma

Jee H. Choe, Tianzhou Yu, Jeremy S. Abramson, Mohamed Abou-el-Enein

Blood Adv (2024) 8 (2): 484–496.

https://doi.org/10.1182/bloodadvances.2023011793

Published: 2024

Includes: Supplemental data

Journal Articles

Nonactivated and IL-7 cultured CD19-specific CAR T cells are enriched in stem cell phenotypes and functionally superior

Siao-Yi Wang, Gina M. Scurti, Annika V. Dalheim, Suzanne Quinn, Patrick J. Stiff, Michael I. Nishimura

Blood Adv (2024) 8 (2): 324–335.

https://doi.org/10.1182/bloodadvances.2023010607

Published: 2024

NARROW

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

Roctavian Gene Therapy for Hemophilia A

Response to: 'In Response to, "Preferences for potential benefits and risks for gene therapy..."'

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

Utilizing Epigenetic Regulators to improve HSC-based lentiviral gene therapy

In Response to, "Preferences for potential benefits and risks for gene therapy in the treatment of sickle cell disease"

Optimizing Liver Health Before and After Gene Therapy for Hemophilia A

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

Mending RAG2: gene editing for treatment of RAG2 deficiency

Long live the red blood cell: biotin tagging in SCD

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

Genetically corrected RAG2 -SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency

Clinical features of neurotoxicity after CD19 CAR T-cell therapy in mantle cell lymphoma

CRISPR/Cas9 deletion of MIR155HG in human T cells reduces incidence and severity of acute GVHD in a xenogeneic model

Choosing the right mouse model: comparison of humanized NSG and NBSGW mice for in vivo HSC gene therapy

Answering the “Doctor, can CAR-T therapy cause cancer?” question in clinic

Cost-effectiveness of second-line lisocabtagene maraleucel in relapsed or refractory diffuse large B-cell lymphoma

Nonactivated and IL-7 cultured CD19-specific CAR T cells are enriched in stem cell phenotypes and functionally superior

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American Society of Hematology

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ASH Publications

American Society of Hematology

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