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Journal Articles

Gene therapy for sickle cell disease: moving from the bench to the bedside

Allistair A. Abraham, John F. Tisdale

Blood (2021) 138 (11): 932–941.

https://doi.org/10.1182/blood.2019003776

Published: 2021

Journal Articles

Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua

Nisha Nair, Dries De Wolf, Phuong Anh Nguyen, Quang Hong Pham, Ermira Samara-Kuko, Jeff Landau, Grant E. Blouse, Marinee K. Chuah, Thierry VandenDriessche

Blood (2021) 137 (21): 2902–2906.

https://doi.org/10.1182/blood.2020006005

Published: 2021

Includes: Supplemental data

Journal Articles

No CpGs for AAVs?

Lindsey A. George

Blood (2021) 137 (6): 721–723.

https://doi.org/10.1182/blood.2020009285

Published: 2021

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

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

Blood (2020) 135 (15): 1219–1231.

https://doi.org/10.1182/blood.2019002350

Published: 2020

Includes: Supplemental data

Journal Articles

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Erica B. Esrick, MD, Maureen Achebe, MD MPH, Myriam Armant, PhD, Pablo Bartolucci, MD PhD, Marioara Felicia Ciuculescu, MD, Heather Daley, Colleen Dansereau, MSN, RN, CPN, Giuseppe Di Caprio, PhD, Bronner Goncalves, MD PhD, Nicolas Hebert, Matthew M Heeney, MD, John M. Higgins, MD PhD, Leslie E. Lehmann, MD, John P Manis, MD, Olivier Negre, PhD, Sarah Nikiforow, MD PhD, Ethan Schonbrun, PhD, David K. Wood, PhD, David A. Williams, MD

Blood (2019) 134 (Supplement_2): LBA-5.

https://doi.org/10.1182/blood-2019-132745

Published: 2019

Journal Articles

C/EBPβ Isoforms Regulate Proliferation and Differentiation of Regenerating Hematopoietic Stem/Progenitor Cells

Atsushi Sato, MD, Asumi Yokota, PhD, Yoshihiro Hayashi, MD PhD, Naoka Kamio, MD, Satoshi Sagai, Taira Maekawa, MD PhD, Hideyo Hirai, MD PhD

Blood (2019) 134 (Supplement_1): 3713.

https://doi.org/10.1182/blood-2019-128026

Published: 2019

Journal Articles

Megakaryocyte Ablation Ameliorates Bone Marrow Fibrosis By Reducing Myofibroblast Differentiation in a Mouse Model of Primary Myelofibrosis

Yutein Chung, PhD, Huiyan Jin, PhD, Claire Trasorras, Francina Gonzalez, Sang Hee Min, MD PhD

Blood (2019) 134 (Supplement_1): 2493.

https://doi.org/10.1182/blood-2019-132186

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

Beyond the Routine CBC: Research CBC Parameters Associated with Myelodysplastic Syndromes and Underlying Mutations

Olga Pozdnyakova, MD PhD, Radu Stefan Niculescu, PhD, Tracey Kroll, BSc, Lisa Golemme, MLS (ASCP), MS, Nolan Raymond, MLS (ASCP), Debra Briggs, MT(ASCP), MS, Annette S. Kim, MD PhD

Blood (2019) 134 (Supplement_1): 5419.

https://doi.org/10.1182/blood-2019-125654

Published: 2019

Journal Articles

BCR-ABL and Src Family Kinases Induce YAP Tyrosine Phosphorylation Resulting in Survivin and Cyclin D1 Expression in Chronic Myeloid Leukemia Cells

Toshiyuki Hori, MD PhD, Kenta Moriyama, MS

Blood (2019) 134 (Supplement_1): 5366.

https://doi.org/10.1182/blood-2019-124643

Published: 2019

Journal Articles

Risk Factors and Outcomes of Patients with Follicular Lymphoma Who Had Histologic Transformation at First Progression after First-Line Immunochemotherapy in the Gallium Study

Carla Casulo, Michael Herold, MD, Wolfgang Hiddemann, MD PhD, Sunil Iyengar, MD, Robert E. Marcus, FRCP, John F. Seymour, MBBS, PhD FRACP, Aino Launonen, MSc, Andrea Knapp, PhD, Tina G Nielsen, MD PhD, Farheen Mir, MBBS, MD

Blood (2019) 134 (Supplement_1): 5268.

https://doi.org/10.1182/blood-2019-123466

Published: 2019

Journal Articles

Activation of JAK2/STAT5 Pathway Reduces Expression Level of DNMT3a in MPN Cell Line

Jie Zhou, Aibin Liang, PhD, Shaoguang Li, Wenjun Zhang, Jianfei FU, MD

Blood (2019) 134 (Supplement_1): 5394.

https://doi.org/10.1182/blood-2019-125434

Published: 2019

Journal Articles

The Effect of Apoptosis Inhibitor Blockade Agents on the Third Generation CD19 CAR T Cells

Mingya Yang, MD, Lei Wang, MD, Ming Ni, Maria-Luisa Schubert, MD, Brigitte Neuber, Angela Hückelhoven-Krauss, Ruben A. Gößmann, Christian Kleist, Volker Eckstein, Carsten Müller-Tidow, MD, Peter Dreger, Michael Schmitt, MD, Anita Schmitt, MD

Blood (2019) 134 (Supplement_1): 5620.

https://doi.org/10.1182/blood-2019-125622

Published: 2019

Journal Articles

Identification of Deubiquitinase OTUD1 As a Novel Player in Resistance of Multiple Myeloma to Bortezomib

Alexander Vdovin, Tomas Jelinek, MD, Matous Hrdinka, PhD, Juli R. Bago, PhD, Tereza Sevcikova, Roman Hajek, Prof, MD PhD, Michal Simicek, PhD

Blood (2019) 134 (Supplement_1): 5526.

https://doi.org/10.1182/blood-2019-127930

Published: 2019

Journal Articles

Examining the Role of CD30 in an Anaplastic Large Cell Lymphoma Mouse Model

Teresa Poggio, Linda Graessel, Stefanie Kreutmair, Cornelius Miething, Dietmar Pfeifer, Geoffroy Andrieux, Melanie Boerries, Marie Follo, Suzanne Turner, Robert Zeiser, MD, Justus Duyster, MD, Anna Lena Illert

Blood (2019) 134 (Supplement_1): 2542.

https://doi.org/10.1182/blood-2019-125589

Published: 2019

Journal Articles

Mutant ASXL1 Disrupts Paraspeckle Formation through Aberrant Interaction with Nono in Hematopoietic Cells

Yamamoto Keita, MD, Susumu Goyama, MD PhD, Shuhei Asada, MD PhD, Takeshi Fujino, MD, Tomofusa Fukuyama, MD PhD, Yosuke Tanaka, MD, Ayana Kon, MD PhD, Yasuhito Nannya, MD PhD, Seishi Ogawa, MD PhD, Reina Onoguchi, PhD, Nobuyoshi Akimitsu, PhD, Toshio Kitamura, MD PhD

Blood (2019) 134 (Supplement_1): 2514.

https://doi.org/10.1182/blood-2019-125472

Published: 2019

Journal Articles

The Translocation t(7;12)(q36;p13) Induces Myeloid Leukemia in Immuno-Compromised but Not Immunocompetent Mice

Ahmed Waraky, PhD MSc, BSc, Anders Östlund, MSc, BSc, Laleh Arabanian, PhD, Tina Nilsson, MSc, BSc, Linda Fogelstrand, Lars Palmqvist, MD PhD

Blood (2019) 134 (Supplement_1): 2707.

https://doi.org/10.1182/blood-2019-123019

Published: 2019

Journal Articles

Dual Inhibition of MDM2 and XPO1 Synergizes to Induce Apoptosis in Acute Myeloid Leukemia Progenitor Cells with Wild-Type TP53 through Nuclear Accumulation of p53 and Suppression of c-Myc

Yuki Nishida, MD PhD, Jo Ishizawa, MD PhD, Vivian Ruvolo, MS, Kensuke Kojima, MD PhD, Rafael Heinz Montoya, Naval G. Daver, MD, Arnaud Lesegretain, Sharon Shacham, PhD MBA, Michael Andreeff, MD PhD

Blood (2019) 134 (Supplement_1): 2556.

https://doi.org/10.1182/blood-2019-126950

Published: 2019

Journal Articles

Induction Related Mortality in Acute Myeloid Leukemia: Multivariate Model of Predictive Score from the Indian Acute Leukemia Research Database (INwARD) of the Hematology Cancer Consortium (HCC)

Blood (2019) 134 (Supplement_1): 2615.

https://doi.org/10.1182/blood-2019-127623

Published: 2019

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Gene therapy for sickle cell disease: moving from the bench to the bedside

Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua

No CpGs for AAVs?

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

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

C/EBPβ Isoforms Regulate Proliferation and Differentiation of Regenerating Hematopoietic Stem/Progenitor Cells

Megakaryocyte Ablation Ameliorates Bone Marrow Fibrosis By Reducing Myofibroblast Differentiation in a Mouse Model of Primary Myelofibrosis

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

Beyond the Routine CBC: Research CBC Parameters Associated with Myelodysplastic Syndromes and Underlying Mutations

BCR-ABL and Src Family Kinases Induce YAP Tyrosine Phosphorylation Resulting in Survivin and Cyclin D1 Expression in Chronic Myeloid Leukemia Cells

Risk Factors and Outcomes of Patients with Follicular Lymphoma Who Had Histologic Transformation at First Progression after First-Line Immunochemotherapy in the Gallium Study

Activation of JAK2/STAT5 Pathway Reduces Expression Level of DNMT3a in MPN Cell Line

The Effect of Apoptosis Inhibitor Blockade Agents on the Third Generation CD19 CAR T Cells

Identification of Deubiquitinase OTUD1 As a Novel Player in Resistance of Multiple Myeloma to Bortezomib

Examining the Role of CD30 in an Anaplastic Large Cell Lymphoma Mouse Model

Mutant ASXL1 Disrupts Paraspeckle Formation through Aberrant Interaction with Nono in Hematopoietic Cells

The Translocation t(7;12)(q36;p13) Induces Myeloid Leukemia in Immuno-Compromised but Not Immunocompetent Mice

Dual Inhibition of MDM2 and XPO1 Synergizes to Induce Apoptosis in Acute Myeloid Leukemia Progenitor Cells with Wild-Type TP53 through Nuclear Accumulation of p53 and Suppression of c-Myc

Induction Related Mortality in Acute Myeloid Leukemia: Multivariate Model of Predictive Score from the Indian Acute Leukemia Research Database (INwARD) of the Hematology Cancer Consortium (HCC)

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