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Journal Articles

A phase 1 study of a novel fully human BCMA-targeting CAR (CT103A) in patients with relapsed/refractory multiple myeloma

Blood (2021) 137 (21): 2890–2901.

https://doi.org/10.1182/blood.2020008936

Published: 2021

Includes: Supplemental data

Journal Articles

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Barbara A. Konkle, Christopher E. Walsh, Miguel A. Escobar, Neil C. Josephson, Guy Young, Annette von Drygalski, Scott W. J. McPhee, R. Jude Samulski, Ivan Bilic, Maurus de la Rosa, Birgit M. Reipert, Hanspeter Rottensteiner, Friedrich Scheiflinger, John C. Chapin, Bruce Ewenstein, Paul E. Monahan

Blood (2021) 137 (6): 763–774.

https://doi.org/10.1182/blood.2019004625

Published: 2021

Includes: Supplemental data

Journal Articles

Mutant U2AF1 S34F and U2AF1 Q157P Induce Distinct RNA Splicing and Hematopoietic Phenotypes In Vivo

Michael O. Alberti, MD PhD, Meng'ou Zhu, BS, Sridhar Nonavinkere Srivatsan, MS, Tanzir Ahmed, PhD, Jin J. Shao, Brian Wadugu, BA, Cara Lunn Shirai, PhD, Matthew J. Walter, MD

Blood (2019) 134 (Supplement_1): 770.

https://doi.org/10.1182/blood-2019-123892

Published: 2019

Journal Articles

Design and Characterization of FLT210, a Potent Next Generation AAV-hFVIII Vector Candidate

Blood (2019) 134 (Supplement_1): 4638.

https://doi.org/10.1182/blood-2019-128490

Published: 2019

Journal Articles

Pulsation Activates Mechanosensitive Piezo1 to Form Long-Term Hematopoietic Stem Cells

Giorgia Scapin, PhD, Dhvanit I Shah, PhDPharmD

Blood (2019) 134 (Supplement_1): 445.

https://doi.org/10.1182/blood-2019-121948

Published: 2019

Journal Articles

Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

Laura Breda, PhD, Valentina Ghiaccio, PhD, Silvia Pires Lourenco, MSc, Danuta Jadwiga Jarocha, PhD, Yasuhiro Ikawa, MD PhD, Naoto Tanaka, PhD, Virginia Guzikowski, Carla Casu, PhD, Kim Smith-Whitley, MD, Janet L. Kwiatkowski, MD MSCE, Stefano Rivella, PhD

Blood (2019) 134 (Supplement_1): 2242.

https://doi.org/10.1182/blood-2019-124043

Published: 2019

Journal Articles

Idh2R140Q Cooperates with NHD13 to Induce Immature T Cell Leukemia By Targeting Early T Cell Progenitors or DN2 Thymocytes

Vijay Negi, PhD, Liat Goldberg, PhD, Yang Jo Chung, PhD, Masahiro Onozawa, MD, Peter D. Aplan, MD

Blood (2019) 134 (Supplement_1): 1346.

https://doi.org/10.1182/blood-2019-128675

Published: 2019

Journal Articles

The Gp1ba-Cre transgenic mouse: a new model to delineate platelet and leukocyte functions

Zoltan Nagy, Timo Vögtle, Mitchell J. Geer, Jun Mori, Silke Heising, Giada Di Nunzio, Ralph Gareus, Alexander Tarakhovsky, Arthur Weiss, Benjamin G. Neel, Guillaume E. Desanti, Alexandra Mazharian, Yotis A. Senis

Blood (2019) 133 (4): 331–343.

https://doi.org/10.1182/blood-2018-09-877787

Published: 2019

Includes: Supplemental data

Journal Articles

TGF-β Signaling in Fetal Mesenchymal Progenitor Cells Plays an Essential Role in the Emergence of Bone Marrow Hematopoietic Niches

Grazia Abou Ezzi, PhD, Teerawit Supakorndej, BS, Jingzhu Zhang, BS, Joseph R. Krambs, BS, Hamza Celik, PhD, Daniel C. Link, MD

Blood (2018) 132 (Supplement 1): 3847.

https://doi.org/10.1182/blood-2018-99-117396

Published: 2018

Journal Articles

NRAS G12V -Mediated Self-Renewal Signaling in Self-Renewing AML Stem Cells.

Marie Lue Antony, PhD, Klara Noble-Orcutt, Karen Sachs, PhD, Anna Khoroshilov, Daniel Chang, Alexandria Hillesheim, Hesham Mohei, Merve Pamukcuoglu, MD, Christine Bergene, David A. Largaespada, PhD, Zohar Sachs, MD PhD

Blood (2018) 132 (Supplement 1): 2626.

https://doi.org/10.1182/blood-2018-99-119448

Published: 2018

Journal Articles

Exon Skipping of AIMP2 and Lymphomagenesis

Doyeun Kim, PhD, Seulki Song, Dae-Yoon Kim, Dongchan Kim, Nam Hoon Kwon, PhD, Byung-Su Kim, MD PhD, Hyun Jung Lee, MD, Hyo Jung Kim, MD PhD, Jung Lim Lee, MD, Sung-Soo Yoon, MD PhD, Yoon Kyung Jeon, MD PhD, Sunghoon Kim, Youngil Koh, MD PhD

Blood (2018) 132 (Supplement 1): 2630.

https://doi.org/10.1182/blood-2018-99-117596

Published: 2018

Journal Articles

The Complete Dependence of Factor IX Padua (R338L) Hyperactivity on Factor VIIIa Cofactor Activity Supports Its Safety As a Transgene for Hemophilia B Gene Therapy

Benjamin J. Samelson-Jones, MD PhD, John D. Finn, PhD, Rodney M. Camire, PhD, Valder R Arruda, MD PhD

Blood (2018) 132 (Supplement 1): 3486.

https://doi.org/10.1182/blood-2018-99-119270

Published: 2018

Journal Articles

Conversion of T-Effector Cells to Immunosuppressive T-Regulatory-like Cells By CRISPR/Cas9-Mediated Integration of a FOXP3 Transgene

Yuchi Honaker, PhD, Yufei Xiang, MD, PhD, Logan Fisher, B.S., Karen Sommer, PhD, Troy R. Torgerson, MD PhD, Andrew M. Scharenberg, MD, David J. Rawlings, MD

Blood (2018) 132 (Supplement 1): 3490.

https://doi.org/10.1182/blood-2018-99-117456

Published: 2018

Journal Articles

Activity of a FIX-Padua Transgene Product in Commonly Used FIX:C One-Stage and Chromogenic Assay Systems Following PF-06838435 (SPK-9001) Gene Delivery

Mary Robinson, PhD, Lindsey A. George, MD, Benjamin J. Samelson-Jones, MD PhD, Valder R Arruda, MD PhD, Katherine A. High, MD, Marcus E Carr, MD PhD, Stefan Tiefenbacher, PhD

Blood (2018) 132 (Supplement 1): 2198.

https://doi.org/10.1182/blood-2018-99-119616

Published: 2018

Journal Articles

A Simplified Method for Transduction and Expansion of T Cells for Clinical Application

Mariam Khalil, Aaron Foster, PhD, Christine Gagliardi, PhD

Blood (2018) 132 (Supplement 1): 4555.

https://doi.org/10.1182/blood-2018-99-119254

Published: 2018

Journal Articles

Ets1 Enhances Context-Dependent Notch1 Activity in T-Cell Leukemia

Anna McCarter, BS, Ran Yan, Amparo Serna Alarcon, Catherine Chang, Erin Kim, Cher Sha, Yiran Liu, Qing Wang, PhD, Luke F. Peterson, PhD, Rork Kuick, Russell Ryan, MD, Moshe Talpaz, MD, Linda Samuelson, PhD, Michael Ostrowski, PhD, Mark Y Chiang, MD PhD

Blood (2018) 132 (Supplement 1): 2595.

https://doi.org/10.1182/blood-2018-99-111376

Published: 2018

Journal Articles

Mice with a Unique Mutator Phenotype Allow Detection of Lymphoid Leukemia Tumor Suppressor Genes

Mianmian Yin, PhD, Timour Baslan, PhD, Amy Freeland, PhD, Toshihiro Matsukawa, PhD, Steven C. Pruitt, PhD, Peter D. Aplan, MD

Blood (2018) 132 (Supplement 1): 1332.

https://doi.org/10.1182/blood-2018-99-115176

Published: 2018

Journal Articles

Evaluation of In Vivo CAR Transgene Levels in Relapsed/Refractory Pediatric and Young Adult ALL and Adult DLBCL Tisagenlecleucel-Treated Patients

Blood (2018) 132 (Supplement 1): 899.

https://doi.org/10.1182/blood-2018-99-116385

Published: 2018

Journal Articles

U2AF1(S34F) Mutant Hematopoietic Cells Require Expression of Wild-Type U2af1 for Survival

Brian Wadugu, BA, Amanda Heard, Joseph Bradley, BS, Matthew Ndonwi, PhD, Jin J Shao, Matthew J. Walter, MD

Blood (2018) 132 (Supplement 1): 941.

https://doi.org/10.1182/blood-2018-99-110836

Published: 2018

Journal Articles

High-Efficiency Lentiviral Genetic Modification of Primary Human Natural Killer Cells

Giacomo Waller, BS, David Allan, Dhana Chinnasamy, MSc, PhD, Mala Chakraborty, PhD, Emily Levy, MS, Michael Hochman, MD, Long Chen, MD PhD, Akkapon Poolcharoen, Georg Aue, Kate Stringaris, MD PhD, Robert Reger, MS, Richard Childs, MD

Blood (2017) 130 (Supplement 1): 5566.

https://doi.org/10.1182/blood.V130.Suppl_1.5566.5566

Published: 2017

NARROW

A phase 1 study of a novel fully human BCMA-targeting CAR (CT103A) in patients with relapsed/refractory multiple myeloma

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Mutant U2AF1 S34F and U2AF1 Q157P Induce Distinct RNA Splicing and Hematopoietic Phenotypes In Vivo

Design and Characterization of FLT210, a Potent Next Generation AAV-hFVIII Vector Candidate

Pulsation Activates Mechanosensitive Piezo1 to Form Long-Term Hematopoietic Stem Cells

Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

Idh2R140Q Cooperates with NHD13 to Induce Immature T Cell Leukemia By Targeting Early T Cell Progenitors or DN2 Thymocytes

The Gp1ba-Cre transgenic mouse: a new model to delineate platelet and leukocyte functions

TGF-β Signaling in Fetal Mesenchymal Progenitor Cells Plays an Essential Role in the Emergence of Bone Marrow Hematopoietic Niches

NRAS G12V -Mediated Self-Renewal Signaling in Self-Renewing AML Stem Cells.

Exon Skipping of AIMP2 and Lymphomagenesis

The Complete Dependence of Factor IX Padua (R338L) Hyperactivity on Factor VIIIa Cofactor Activity Supports Its Safety As a Transgene for Hemophilia B Gene Therapy

Conversion of T-Effector Cells to Immunosuppressive T-Regulatory-like Cells By CRISPR/Cas9-Mediated Integration of a FOXP3 Transgene

Activity of a FIX-Padua Transgene Product in Commonly Used FIX:C One-Stage and Chromogenic Assay Systems Following PF-06838435 (SPK-9001) Gene Delivery

A Simplified Method for Transduction and Expansion of T Cells for Clinical Application

Ets1 Enhances Context-Dependent Notch1 Activity in T-Cell Leukemia

Mice with a Unique Mutator Phenotype Allow Detection of Lymphoid Leukemia Tumor Suppressor Genes

Evaluation of In Vivo CAR Transgene Levels in Relapsed/Refractory Pediatric and Young Adult ALL and Adult DLBCL Tisagenlecleucel-Treated Patients

U2AF1(S34F) Mutant Hematopoietic Cells Require Expression of Wild-Type U2af1 for Survival

High-Efficiency Lentiviral Genetic Modification of Primary Human Natural Killer Cells

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American Society of Hematology

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American Society of Hematology

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