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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

Sickle Cell Hemoglobin Impairs Lysosomal Processing in Macrophages Leading to Inflammasome Activation and Inflammation

Marina Jerebtsova, PhD, Xiaomei Niu, MD, Sharmin Diaz, NP, Namita Kumari, PhD, Sergei Nekhai, PhD

Blood (2019) 134 (Supplement_1): 979.

https://doi.org/10.1182/blood-2019-130741

Published: 2019

Journal Articles

Fibrinogen Activates FAK to Promote the Colorectal Adenocarcinoma Growth

Balkrishan Sharma, PhD, Duaa Mureb, BS, Sumit Murab, PhD, Leah A. Rosenfeldt, BS, Brenton J Francisco, MD, Alexander A Boucher, MD, Rachel Cantrell, BS, Jacob Mast, Matthew J. Flick, PhD, Patrick W Whitlock, MD, Joseph S. Palumbo, MD

Blood (2019) 134 (Supplement_1): 1111.

https://doi.org/10.1182/blood-2019-130497

Published: 2019

Journal Articles

Pediatric Patient with Hypodysfibrinogenemia Presenting with an Unprovoked Portal Vein Thrombosis

Brenton J Francisco, MD, Balkrishan Sharma, PhD, Phillip A Owens, PhD, Hannah Russell, Joseph S. Palumbo, MD

Blood (2019) 134 (Supplement_1): 1145.

https://doi.org/10.1182/blood-2019-123760

Published: 2019

Journal Articles

Fibg 390-396A Limits Metastatic Potential Via a Mechanism Independent of Fibrin-Myeloid Cell Interactions, but Prospectively through the Disruption of FXIII-Mediated Fibrin Crosslinking

Brenton J Francisco, MD, Balkrishan Sharma, PhD, Leah A. Rosenfeldt, BS, Duaa Mureb, BS, Joseph S. Palumbo, MD

Blood (2019) 134 (Supplement_1): 2385.

https://doi.org/10.1182/blood-2019-131644

Published: 2019

Journal Articles

NTBI Is Transiently Generated from Two Compartments during a Single Dose of Intravenous Iron - a Randomized Controlled Clinical Study

Maciej W Garbowski, MD PhD, Sukhvinder S Bansal, PhD, John B. Porter, MA MDFRCP FRCPath, Susanna Burckhardt, PhD, Robert Hider, PhD

Blood (2019) 134 (Supplement_1): 2234.

https://doi.org/10.1182/blood-2019-125618

Published: 2019

Journal Articles

NKTR-255, a Polymer-Conjugated IL-15 Enhances Antibody-Dependent Cellular Cytotoxicity Mediated By NK Cells in a B Cell Lymphoma Model

Takahiro Miyazaki, Saul Kivimäe, Marlene Hennessy, Rhoneil Pena, Phi Quach, Andrew Moffet, Wildaliz Nieves, Ping Zhang, Mario Q. Marcondes, MD PhD, Loui Madakamutil, Jonathan Zalevsky

Blood (2019) 134 (Supplement_1): 5302.

https://doi.org/10.1182/blood-2019-127771

Published: 2019

Journal Articles

Combination of NKTR-255, a Polymer Conjugated Human IL-15, with CD19 CAR T Cell Immunotherapy in a Preclinical Lymphoma Model

Cassie Chou, MD PhD, Simon Fraessle, Rachel Steinmetz, BSc, Reed M. Hawkins, BS, Tinh-Doan Phi, BS, Dirk Busch, Takahiro Miyazaki, Mario Q. Marcondes, MD PhD, Stanley R. Riddell, MD, Cameron J. Turtle, MBBS, PhD

Blood (2019) 134 (Supplement_1): 2866.

https://doi.org/10.1182/blood-2019-125960

Published: 2019

Journal Articles

Complementary Base Editing Approaches for the Treatment of Sickle Cell Disease and Beta Thalassemia

Ling Lin, PhD, Adrian P. Rybak, PhD, Conrad Rinaldi, M.S., Jonathan Yen, PhD, Yanfang Fu, PhD, Elsie Akrawi, Sarah Smith, Scott J. Haskett, M.S., Minerva E. Sanchez, Yeh-Chuin Poh, PhD, Michael S. Packer, PhD, Nicole M. Gaudelli, PhD, Manmohan Singh, PhD, Giuseppe Ciaramella, PhD, B.Sc., Dana N. Levasseur, PhD

Blood (2019) 134 (Supplement_1): 3352.

https://doi.org/10.1182/blood-2019-126710

Published: 2019

Journal Articles

Improvement in Red Blood Cell Physiology in Children with Sickle Cell Anemia Receiving Voxelotor

Satheesh Chonat, MD, Earl Fields, Hannah Baratz, Amanda Watt, Mira Pochron, PhD, Sandy Dixon, Margaret Tonda, PharmD, Joshua Lehrer-Graiwer, MD MPhil, FACC, Clark Brown, MD PhD, David R. Archer, PhD

Blood (2019) 134 (Supplement_1): 2281.

https://doi.org/10.1182/blood-2019-129351

Published: 2019

Journal Articles

A Phase 1, Open-Label, Multi-Center, Dose Escalation and Dose Expansion Study of NKTR-255 As a Single Agent in Relapsed or Refractory Hematologic Malignancies and in Combination with Daratumumab As a Salvage Regimen for Multiple Myeloma

Nina D. Shah, MD, Cameron J. Turtle, MBBS, PhD, Andrew J. Cowan, MD, Julio C. Chavez, MD, Lihua E Budde, MD PhD, Mario Q. Marcondes, MD PhD, Zachary Lee, PharmD, Wei Lin, MD, Jonathan Zalevsky, Mary Tagliaferri, MD, Krina K. Patel, MDMSc

Blood (2019) 134 (Supplement_1): 4459.

https://doi.org/10.1182/blood-2019-122494

Published: 2019

Journal Articles

PP-14, a Novel Structurally-Enhanced Antisickling Allosteric Hemoglobin Effector, Increases Oxygen Affinity and Disrupts Hemoglobin S Polymer Formation

Martin K. Safo, PhD, Piyusha P Pagare, PhD, Mohini Ghatge, PhD, Carla Casu, PhD, Valentina Ghiaccio, PhD, Nancy Anabaraonye, Xiaomeng Xu, MS, Stefano Rivella, PhD, Patrick C. Hines, MD PhD, Ke Liu, PhD, Yan Zhang, PhD, Jürgen Venitz, MD PhD, Osheiza Abdulmalik, DVM

Blood (2019) 134 (Supplement_1): 73.

https://doi.org/10.1182/blood-2019-128646

Published: 2019

Journal Articles

Restoring Innate and Adaptive Immune Repertoire in Multiple Myeloma for Therapeutic Application

Rafael Alonso Fernández, MD, Laetitia Pierre-Louis, Rao Prabhala, Yan Xu, MD, Joaquin Martinez-Lopez, MD PhD, Takahiro Miyazaki, Mehmet Kemal Samur, PhD, Loui Madakamutil, Mariateresa Fulciniti, PhD, Nikhil C. Munshi, MD

Blood (2019) 134 (Supplement_1): 4398.

https://doi.org/10.1182/blood-2019-128767

Published: 2019

Journal Articles

Regulation of Coagulation by Polyphosphate

Nicola J. Mutch, BSc, PhD

Blood (2019) 134 (Supplement_1): SCI-18.

https://doi.org/10.1182/blood-2019-121062

Published: 2019

Journal Articles

Bio-Inspired Artificial Red Blood Cell: Design, Pre-Clinical Results and Novel Indications

Allan Doctor, MD

Blood (2019) 134 (Supplement_1): SCI-4.

https://doi.org/10.1182/blood-2019-121115

Published: 2019

Journal Articles

Spatial Structure of Plasma Coagulation Factor XIII and Changes with Activation

Anna D. Protopopova, PhD, Andrea Ramirez, Rustem I. Litvinov, John W. Weisel, PhD

Blood (2018) 132 (Supplement 1): 19.

https://doi.org/10.1182/blood-2018-99-112806

Published: 2018

Journal Articles

Methotrexate-Loaded Biodegradable Polymeric Micelles for Lymphoma Therapy in Mouse Model

Mengyao Wang, MD, Ying Qu, PhD, Zhiyong Qian, PhD, Ting Niu, MD PhD

Blood (2018) 132 (Supplement 1): 4181.

https://doi.org/10.1182/blood-2018-99-119151

Published: 2018

Journal Articles

Drug-Free Macromolecular Therapeutics Induce Apoptosis in Cells Isolated from Patients with B Cell Malignancies with Enhanced Apoptosis Induction By Pretreatment with Gemcitabine

Jiawei Wang, Lian Li, PhD, Jiyuan Yang, PhD, Phillip M. Clair, MS, Martha Glenn, MD, Deborah M. Stephens, D. Christopher Radford, PhDCandidate, Ken M. Kosak, Michael W. Deininger, MD PhD, Paul J Shami, MD, Jindřich Kopeček, PhD

Blood (2018) 132 (Supplement 1): 4426.

https://doi.org/10.1182/blood-2018-99-112001

Published: 2018

Journal Articles

Soluble Guanylate Cyclase Modulators Effect Fetal Hemoglobin Levels through a Non-cGMP-Protein Kinase G Signaling Pathway

Bin Sun, PhD, Valerie Miller, MS, Karin P Potoka, MD, Yukio Nakamura, MD PhD, Mark T Gladwin, MD, Grant C. Bullock, MD PhD

Blood (2018) 132 (Supplement 1): 1060.

https://doi.org/10.1182/blood-2018-99-120306

Published: 2018

Journal Articles

A Method to Fluorescently Label the CRISPR/Cas9-gRNA RNP Complexes Enables Enrichment of Clinical-Grade Gene-Edited Primary Hematopoietic Stem Cells and iPSCs

Masoud Nasri, Perihan Mir, Benjamin Dannenmann, Diana Amend, Yun Xu, Anna Solovyeva, Sylwia Stefanczyk, Maksim Klimiankou, Cornelia Zeidler, Lothar Kanz, Karl Welte, Julia Skokowa, MDPhD

Blood (2018) 132 (Supplement 1): 1108.

https://doi.org/10.1182/blood-2018-99-114844

Published: 2018

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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

Sickle Cell Hemoglobin Impairs Lysosomal Processing in Macrophages Leading to Inflammasome Activation and Inflammation

Fibrinogen Activates FAK to Promote the Colorectal Adenocarcinoma Growth

Pediatric Patient with Hypodysfibrinogenemia Presenting with an Unprovoked Portal Vein Thrombosis

Fibg 390-396A Limits Metastatic Potential Via a Mechanism Independent of Fibrin-Myeloid Cell Interactions, but Prospectively through the Disruption of FXIII-Mediated Fibrin Crosslinking

NTBI Is Transiently Generated from Two Compartments during a Single Dose of Intravenous Iron - a Randomized Controlled Clinical Study

NKTR-255, a Polymer-Conjugated IL-15 Enhances Antibody-Dependent Cellular Cytotoxicity Mediated By NK Cells in a B Cell Lymphoma Model

Combination of NKTR-255, a Polymer Conjugated Human IL-15, with CD19 CAR T Cell Immunotherapy in a Preclinical Lymphoma Model

Complementary Base Editing Approaches for the Treatment of Sickle Cell Disease and Beta Thalassemia

Improvement in Red Blood Cell Physiology in Children with Sickle Cell Anemia Receiving Voxelotor

A Phase 1, Open-Label, Multi-Center, Dose Escalation and Dose Expansion Study of NKTR-255 As a Single Agent in Relapsed or Refractory Hematologic Malignancies and in Combination with Daratumumab As a Salvage Regimen for Multiple Myeloma

PP-14, a Novel Structurally-Enhanced Antisickling Allosteric Hemoglobin Effector, Increases Oxygen Affinity and Disrupts Hemoglobin S Polymer Formation

Restoring Innate and Adaptive Immune Repertoire in Multiple Myeloma for Therapeutic Application

Regulation of Coagulation by Polyphosphate

Bio-Inspired Artificial Red Blood Cell: Design, Pre-Clinical Results and Novel Indications

Spatial Structure of Plasma Coagulation Factor XIII and Changes with Activation

Methotrexate-Loaded Biodegradable Polymeric Micelles for Lymphoma Therapy in Mouse Model

Drug-Free Macromolecular Therapeutics Induce Apoptosis in Cells Isolated from Patients with B Cell Malignancies with Enhanced Apoptosis Induction By Pretreatment with Gemcitabine

Soluble Guanylate Cyclase Modulators Effect Fetal Hemoglobin Levels through a Non-cGMP-Protein Kinase G Signaling Pathway

A Method to Fluorescently Label the CRISPR/Cas9-gRNA RNP Complexes Enables Enrichment of Clinical-Grade Gene-Edited Primary Hematopoietic Stem Cells and iPSCs

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