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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

Next Generation NKG2D-based CAR T-cells (CYAD-02): Co-expression of a Single shRNA Targeting MICA and MICB Improves Cell Persistence and Anti-Tumor Efficacy in vivo

Martina Fontaine, PhD, Benjamin Demoulin, PhD, Simon Bornschein, PhD, Susanna Raitano, PhD, Steve Lenger, Hidevaldo Machado, Jonathan D Moore, Panagiota A. Sotiropoulou, PhD, David E Gilham, PhD

Blood (2019) 134 (Supplement_1): 3931.

https://doi.org/10.1182/blood-2019-129998

Published: 2019

Journal Articles

Identification of T-Cell Receptors Specific to Antigens Presented By HLA-B4002 and B5401 in Acquired Aplastic Anemia

Kohei Hosokawa, MD PhD, Eiji Kobayashi, PhD, Yoshiki Akatsuka, MD PhD, Luis Espinoza, MD PhD, Noriharu Nakagawa, MD, Tanabe Mikoto, MD, Noriaki Tsuji, MD, Takeshi Yoroidaka, MD, Hiroki Mizumaki, MD, Thi Mai Anh Nguyen, MD, Takamasa Katagiri, PhD, Kiyomi Shitaoka, PhD, Hiroshi Hamana, PhD, Hiroyuki Kishi, PhD, Shinji Nakao, MD PhD

Blood (2019) 134 (Supplement_1): 3751.

https://doi.org/10.1182/blood-2019-125685

Published: 2019

Journal Articles

NUP98-HOXA9 -Initiates Molecular and Biologic Features of Disease Progression in a Humanized Model of CML

Ivan Sloma, PharmD, PhD, Philip Beer, PhD FRCPath, MRCP, Christophe Desterke, PhD, Elizabeth Bulaeva, Misha Bilenky, Michelle Moksa, Kamini Raghuram, Cedric Brimacombe, Karen Lambie, Ali G Turhan, MD PhD, Orianne Wagner-Ballon, MD PhD, Philippe Gaulard, MD PhD, R. Keith Humphries, MD PhD, Martin Hirst, PhD BS, Connie J. Eaves, PhD

Blood (2019) 134 (Supplement_1): 4139.

https://doi.org/10.1182/blood-2019-127839

Published: 2019

Journal Articles

Overcoming CD19 Antigen Loss in B-Cell Malignancies with CAR T Cells Targeting BAFF-R

Hong Qin, MD PhD, Zhenyuan Dong, PhD, Xiuli Wang, PhD, Wesley Cheng, BS, Diane Lynne Smith, PhD, Joo Y Song, MD, Ibrahim Aldoss, MD, Stephen J Forman, MD, Larry W Kwak, MD PhD

Blood (2019) 134 (Supplement_1): 3871.

https://doi.org/10.1182/blood-2019-129228

Published: 2019

Journal Articles

Phase 1 Study of on Site Manufactured Anti-CD19 CAR-T Cells: Responses in Subjects with Rapidly Progressive Refractory Lymphomas

Blood (2019) 134 (Supplement_1): 4074.

https://doi.org/10.1182/blood-2019-131300

Published: 2019

Journal Articles

Targeted Delivery of Nanocarrier-Conjugated Doxorubicin to Widen the Therapeutic Window of the Most Active Drug in Lymphoma Therapeutics

Artavazd Arumov, BSc, Piumi Y. Liyanage, BS, Asaad Trabolsi, MD, Lingxiao Li, PhD, Evan R. Roberts, BS, Daniel Bilbao, PhD, Roger M. LeBlanc, PhD, Jonathan H. Schatz, MD

Blood (2019) 134 (Supplement_1): 4061.

https://doi.org/10.1182/blood-2019-122599

Published: 2019

Journal Articles

Surface Density of CAR Molecules May Modulate the Kinetics of CAR-T Cells In Vivo

Jianqiang Li, PhD, Jin-Yuan Ho, PhD, Lin Wang, Xing Niu, Min Ba, Qinglong Wang, Junfang Yang, MD, Xian Zhang, MD, Dandan Chen, Peihua Lu

Blood (2019) 134 (Supplement_1): 54.

https://doi.org/10.1182/blood-2019-125467

Published: 2019

Journal Articles

DNA Polymerases Pol θ/Pol η Involved in Error-Prone DNA Repair Are Highly Expressed in Multiple Myeloma and Upregulated By DNA Damage

Blood (2019) 134 (Supplement_1): 4364.

https://doi.org/10.1182/blood-2019-125163

Published: 2019

Journal Articles

Prediction of High-Risk Group of Primary Refractory Diffuse Large B-Cell Lymphoma (DLBCL) Patients Using a CT-Based Radiomics Model with Machine Learning

Raoul Santiago, MDMSc, Johanna Ortiz Jimenez, MD, Reza Forghani, MD PhD, Nikesh Muthukrishnan, MEng, Olivier Del Corpo, BSC, MSc, Shairabi Karthigesu, Muhammad Yahya Haider, Caroline Reinhold, MD MSc, Sarit Assouline, MD

Blood (2019) 134 (Supplement_1): 4136.

https://doi.org/10.1182/blood-2019-126745

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

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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

Next Generation NKG2D-based CAR T-cells (CYAD-02): Co-expression of a Single shRNA Targeting MICA and MICB Improves Cell Persistence and Anti-Tumor Efficacy in vivo

Identification of T-Cell Receptors Specific to Antigens Presented By HLA-B4002 and B5401 in Acquired Aplastic Anemia

NUP98-HOXA9 -Initiates Molecular and Biologic Features of Disease Progression in a Humanized Model of CML

Overcoming CD19 Antigen Loss in B-Cell Malignancies with CAR T Cells Targeting BAFF-R

Phase 1 Study of on Site Manufactured Anti-CD19 CAR-T Cells: Responses in Subjects with Rapidly Progressive Refractory Lymphomas

Targeted Delivery of Nanocarrier-Conjugated Doxorubicin to Widen the Therapeutic Window of the Most Active Drug in Lymphoma Therapeutics

Surface Density of CAR Molecules May Modulate the Kinetics of CAR-T Cells In Vivo

DNA Polymerases Pol θ/Pol η Involved in Error-Prone DNA Repair Are Highly Expressed in Multiple Myeloma and Upregulated By DNA Damage

Prediction of High-Risk Group of Primary Refractory Diffuse Large B-Cell Lymphoma (DLBCL) Patients Using a CT-Based Radiomics Model with Machine Learning

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

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