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Journal Articles

Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells

Suk See De Ravin, Julie Brault, Ronald J. Meis, Siyuan Liu, Linhong Li, Mara Pavel-Dinu, Cicera R. Lazzarotto, Taylor Liu, Sherry M. Koontz, Uimook Choi, Colin L. Sweeney, Narda Theobald, GaHyun Lee, Aaron B. Clark, Sandra S. Burkett, Benjamin P. Kleinstiver, Matthew H. Porteus, Shengdar Tsai, Douglas B. Kuhns, Gary A. Dahl, Stephen Headey, Xiaolin Wu, Harry L. Malech
Journal: Blood
Blood (2021) 137 (19): 2598–2608.
https://doi.org/10.1182/blood.2020008503
Published: 2021
Includes: Supplemental data
Journal Articles

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

Clinical Trials & Observations
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
Journal: Blood
Blood (2021) 137 (6): 763–774.
https://doi.org/10.1182/blood.2019004625
Published: 2021
Includes: Supplemental data
Journal Articles

RNA-Seq Based Immunoglobulin Repertoire Analysis of Normal Plasma Cells Generated in an in Vitro B Cell Differentiation System

Renee C Tschumper, Collin A Osborne, MS, Pritha Chanana, MS, Jaime I Davila, PhD, Denise K Walters, PhD BSc, Dominique B Hoelzinger, PhD, Diane F Jelinek, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1051.
https://doi.org/10.1182/blood-2019-127327
Published: 2019
Journal Articles

Toll-like Receptor 9 Activation Accelerates Inhibitor Formation in Response to Factor VIII

Radoslaw Kaczmarek, Alexandra Sherman, DVM, Moanaro Biswas, PhD, Roland W Herzog, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1113.
https://doi.org/10.1182/blood-2019-125348
Published: 2019
Journal Articles

Targeting MiR-126 Blocks Progression of Myelodysplastic Syndrome and Transformation into Acute Myeloid Leukemia

Huafeng Wang, MD, Jie Sun, PhD, Dandan Zhao, MD, Hongyan Tong, Herman Wu, BS, Xia Li, PhD, Yingwan Luo, MD, Dan Dong, PhD, Tinisha McDonald, MS, Anthony Stein, MD, Monzr M. Al Malki, MD, Flavia Pichiorri, PhD, Nadia Carlesso, MD, Ya-Huei Kuo, PhD, Bin Zhang, PhD, Guido Marcucci, MD, Ling Li, PhD, Jie Jin, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1707.
https://doi.org/10.1182/blood-2019-130375
Published: 2019
Journal Articles

Anti-MiR-126 Therapy for Inv(16) Acute Myeloid Leukemia

Lianjun Zhang, PhD, Le Xuan Truong Nguyen, PhD, Dijiong Wu, MD, Casey J Brewer, PhD, Xin He, PhD, Man Li, PhD, Dandan Zhao, MD, Guerry Jeanne Cook, PhD, Jing Qi, PhD, Herman Wu, BS, Bin Zhang, PhD, Ling Li, PhD, Marcin Kortylewski, PhD, Guido Marcucci, MD, Ya-Huei Kuo, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3914.
https://doi.org/10.1182/blood-2019-123468
Published: 2019
Journal Articles

Molecular Characterization and Novel Therapeutic Strategy for X-Linked Sideroblastic Anemia Associated with ALAS2 Missense Variants

Koya Ono, MD, Tohru Fujiwara, MD PhD, Kei Saito, MD PhD, Chie Suzuki, Noriyuki Takahashi, Koichi Onodera, MD PhD, Satoshi Ichikawa, MD PhD, Noriko Fukuhara, MD PhD, Yasushi Onishi, MD PhD, Hisayuki Yokoyama, MD PhD, Yukio Nakamura, MD PhD, Hideo Harigae, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3530.
https://doi.org/10.1182/blood-2019-124810
Published: 2019
Journal Articles

Targeted Marrow Radiation (TMI) Improves Therapeutic Efficacy of STAT3 Decoy Molecules By Augmenting Its Delivery and Immune Modulation in an AML Mouse Model

Srideshikan Sargur Madabushi, PhD, Darren Zuro, Yu-Lin SU, PhD, Paresh Vishwasrao, MSc, Jamison Brooks, B.S, Liliana E Parra, James F Sanchez, PhD, Jeffrey Wong, MD, Marcin Kortylewski, PhD, Susanta Hui, PhD, DABR.
Journal: Blood
Blood (2019) 134 (Supplement_1): 3929.
https://doi.org/10.1182/blood-2019-127470
Published: 2019
Journal Articles

Mutated MYD88 Regulates HCK Pro-Survival Signaling through JunB in MYD88 Mutated B-Lymphoma Cells

Xia Liu, MD, Jiaji G Chen, Manit Munshi, MS, Lian Xu, MS, Amanda Kofides, Nicholas Tsakmaklis, Maria Demos, Maria Luisa Guerrera, MD, Gloria G Chan, MS, Cristina Jimenez, PhD, Zachary R Hunter, PhD, Christopher Patterson, Kirsten Meid, MPH, Andrew Keezer, BS, Jorge J. Castillo, MD, Steven P Treon, MD PhD FRCP, Guang Yang, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3778.
https://doi.org/10.1182/blood-2019-130885
Published: 2019
Journal Articles

Comparative Studies Reveal Robust HbF Induction By Editing of HBG1/2 Promoters or BCL11A Erythroid-Enhancer in Human CD34+ Cells but That BCL11A Erythroid-Enhancer Editing Is Associated with Selective Reduction in Erythroid Lineage Reconstitution in a Xenotransplantation Model

Kai-Hsin Chang, PhD, Minerva Sanchez, Jack Heath, Edouard deDreuzy, PhD, Scott Haskett, MS, Abigail Vogelaar, Kiran Gogi, Jen Da Silva, Tongyao Wang, Andrew Sadowski, Gregory Gotta, Jamaica Siwak, Ramya Viswanathan, Katherine Loveluck, Hoson Chao, Eric Tillotson, Aditi Chalishazar, MS, Abhishek Dass, Frederick Ta, Emily Brennan, Diana Tabbaa, Eugenio Marco, PhD, John A Zuris, PhD, Deepak Reyon, PhD, Meltem Isik, PhD, Ari Friedland, PhD, Terence Ta, PhD, Fred Harbinski, Georgia Giannoukos, PhD, Sandra Teixeira, PhD, Christopher J Wilson, PhD, Charlie Albright, PhD, Haiyan Jiang, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 409.
https://doi.org/10.1182/blood-2018-99-114780
Published: 2018
Journal Articles

Investigating Potential Mechanism(s) By Which ASO-Based Drugs Cause Thrombocytopenia

Martina H. Lundberg Slingsby, PhD, Genevieve Couldwell, Prakrith Vijey, Brooke E Terkovich, Leila Noetzli, PhD, Ross Okazaki, Jonathan N. Thon, PhD, Scott P. Henry, PhD, Padmakumar Narayanan, DVM, PhD, Joseph E. Italiano, Jr., PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 3747.
https://doi.org/10.1182/blood-2018-99-116590
Published: 2018
Journal Articles

STAT3 Inhibition Enables TLR9-Driven Differentiation of Cbfb/Myh11 acute Myeloid Leukemia Cells to Antigen-Presenting Cell Phenotype In Vivo

Marcin Kortylewski, PhD, Priyanka Duttagupta, PhD, Yu-Lin SU, PhD, Tomasz Adamus, Guido Marcucci
Journal: Blood
Blood (2018) 132 (Supplement 1): 4070.
https://doi.org/10.1182/blood-2018-99-119771
Published: 2018
Journal Articles

A Multi-Drug Tolerant Phenotype Is Induced By Diverse Microenvironmental Agonists in Chronic Lymphocytic Leukemia and Mantle Cell Lymphoma

Kallesh D. Jayappa, DVM, PhD, Konrad J. Cios, BSc (2019), Vicki L. Gordon, BSc, Puja C. Arora, MD, Timothy P. Bender, PhD, Michael E. Williams, MD, Craig A. Portell, MD, Michael J. Weber, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 4120.
https://doi.org/10.1182/blood-2018-99-119521
Published: 2018
Journal Articles

Gene Editing of BTK in Acute Myeloid Leukaemia Using CRISPR-Cas9

Melanie Maerken, MS, BSc, David MacEwan, PhD, Nicholas Harper, PhD, Joseph R Slupsky, PhD, Adam Linley, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 3952.
https://doi.org/10.1182/blood-2018-99-113804
Published: 2018
Journal Articles

In Situ Vaccination with Cpg and Anti-OX40 Antibody: Preclinical Optimization for Clinical Translation

Idit Sagiv Barfi, PhD, Debra K. Czerwinski, BA, Tanaya Shree, MD PhD BA, Ronald Levy, MD
Journal: Blood
Blood (2018) 132 (Supplement 1): 2943.
https://doi.org/10.1182/blood-2018-99-117872
Published: 2018
Journal Articles

CpG Oligodeoxynucleotide-Induced Apoptosis of B-Lineage Acute Lymphoblastic Leukemia Is through C-MYC/P53/BAX Signaling Pathway

Ling Bai, Wei Chen, MD PhD, Wei Li, MD, Lei Zhou, Chao Niu, Wei Han, Jiuwei Cui
Journal: Blood
Blood (2018) 132 (Supplement 1): 2216.
https://doi.org/10.1182/blood-2018-99-116556
Published: 2018
Journal Articles

Targeted Delivery of CpG-Mir-146a Mimic Oligonucleotides As a Therapeutic Strategy to Reduce NF-Κb-Mediated Pathogenic Inflammation and Myeloid Leukemia Progression

Yu-Lin SU, PhD, Zhuoran Zhang, PhD, Mati Mann, PhD, Xiuli Wang, PhD, Le Xuan Truong Nguyen, PhD, Bin Zhang, PhD, Ling Li, PhD, Piotr Swiderski, PhD, Mark Boldin, MD PhD, Stephen J Forman, Guido Marcucci, Marcin Kortylewski, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 3501.
https://doi.org/10.1182/blood-2018-99-115637
Published: 2018
Journal Articles

Drug Combinations Co-Targeting Myeloid Cell Leukemia-1 (Mcl-1) Protein Can Overcome Microenvironmentally-Induced Multi-Drug Tolerance in Non-Hodgkin Lymphomas

Kallesh D. Jayappa, DVM, PhD, Craig A. Portell, MD, Vicki L. Gordon, BSc, Goutham Narla, MD PhD, Timothy P. Bender, PhD, Michael E. Williams, MD, Michael J. Weber, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 2649.
https://doi.org/10.1182/blood-2018-99-116759
Published: 2018
Journal Articles

Innate Immune Activation Via TLR9 May Enhance Antibody Formation Against Factors VIII and IX in Protein and Gene Therapy

Roland W Herzog, PhD, Alexandra Sherman, DVM, Brad E. Hoffman, PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 3651.
https://doi.org/10.1182/blood.V130.Suppl_1.3651.3651
Published: 2017
Journal Articles

Eradication of Systemic Lymphoma By Local Immunotherapy

Idit Sagiv Barfi, PhD, Debra K. Czerwinski, BA, Ronald Levy, MD
Journal: Blood
Blood (2017) 130 (Supplement 1): 113.
https://doi.org/10.1182/blood.V130.Suppl_1.113.113
Published: 2017