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Journal Articles

Nintedanib targets KIT D816V neoplastic cells derived from induced pluripotent stem cells of systemic mastocytosis

Marcelo A. S. Toledo, Malrun Gatz, Stephanie Sontag, Karoline V. Gleixner, Gregor Eisenwort, Kristina Feldberg, Ahmed E. I. Hamouda, Frederick Kluge, Riccardo Guareschi, Giulia Rossetti, Antonio S. Sechi, Olli M. J. Dufva, Satu M. Mustjoki, Angela Maurer, Herdit M. Schüler, Roman Goetzke, Till Braunschweig, Anne Kaiser, Jens Panse, Mohamad Jawhar, Andreas Reiter, Frank Hilberg, Peter Ettmayer, Wolfgang Wagner, Steffen Koschmieder, Tim H. Brümmendorf, Peter Valent, Nicolas Chatain, Martin Zenke
Journal: Blood
Blood (2021) 137 (15): 2070–2084.
DOI: https://doi.org/10.1182/blood.2019004509
Published: 2021
Includes: Supplemental data
Journal Articles

Analysis of disease model iPSCs derived from patients with a novel Fanconi anemia–like IBMFS ADH5/ALDH2 deficiency

Anfeng Mu, Asuka Hira, Akira Niwa, Mitsujiro Osawa, Kenichi Yoshida, Minako Mori, Yusuke Okamoto, Kazuko Inoue, Keita Kondo, Masato T. Kanemaki, Tomonari Matsuda, Etsuro Ito, Seiji Kojima, Tatsutoshi Nakahata, Seishi Ogawa, Keigo Tanaka, Keitaro Matsuo, Megumu K. Saito, Minoru Takata
Journal: Blood
Blood (2021) 137 (15): 2021–2032.
DOI: https://doi.org/10.1182/blood.2020009111
Published: 2021
Includes: Supplemental data
Journal Articles

Pluripotent stem cell–derived NK cells with high-affinity noncleavable CD16a mediate improved antitumor activity

Huang Zhu, Robert H. Blum, Ryan Bjordahl, Svetlana Gaidarova, Paul Rogers, Tom Tong Lee, Ramzey Abujarour, Gregory B. Bonello, Jianming Wu, Pei-Fang Tsai, Jeffrey S. Miller, Bruce Walcheck, Bahram Valamehr, Dan S. Kaufman
Journal: Blood
Blood (2020) 135 (6): 399–410.
DOI: https://doi.org/10.1182/blood.2019000621
Published: 2020
Includes: Multimedia, Supplemental data
Journal Articles

Efficient Ex Vivo Expansion of Highly Purified Human Umbilical Cord Blood-Derived Very Small CD34 + lin - CD45 - Stem Cells into Functional Endothelial Cells in Vitro in Chemically Identified, Feeder Layer-Free Medium Supplemented with Nicotinamide

Alison Domingues, MS, Kamila Bujko, MS, Magdalena Kucia, PhD DSci, Janina Ratajczak, MD PhD DSci, Mariusz Z Ratajczak, MD PhD DSc
Journal: Blood
Blood (2019) 134 (Supplement_1): 4882.
DOI: https://doi.org/10.1182/blood-2019-125819
Published: 2019
Journal Articles

Investigation of New Therapeutic Compounds for Juvenile Myelomonocytic Leukemia Using Induced Pluripotent Stem Cells with Stably Activated Ras Pathway

Lisa Maria Kuhn, Cyrill Schipp, PhD, Daniel Hein, PhD, Bianca Killing, Nan Qin, PhD, Vera H Jepsen, M.Sc., Heinz Ahlert, MSc, BSc, Jasmin Bartl, PhD, Viktoria Marquardt, M.Sc., David Pauck, Kai Völtzke, M.Sc., Jonathan Lim, PhD, Gabriel Leprivier, PhD, Marc Remke, MD, Sanil Bhatia, PhD, Thorsten Rosenbaum, MD, Arndt Borkhardt, MD, Ute Fischer, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4651.
DOI: https://doi.org/10.1182/blood-2019-131406
Published: 2019
Journal Articles

In silico Prediction Followed By in Vitro validation Identifies the Survivin Inhibitor YM155 As a Potent Secondary Drug Against Pi-Resistant Myeloma

Harish Kumar, PhD, Ujjal Kumar Mukherjee, PhD, Li Chen, PhD, Amit Kumar Mitra, PhD MSc, BSc
Journal: Blood
Blood (2019) 134 (Supplement_1): 5543.
DOI: https://doi.org/10.1182/blood-2019-131923
Published: 2019
Journal Articles

Disease Modeling of Severe Congenital Neutropenia Using CRISPR/Cas9 Gene Correction or Knockout of ELANE in Patients Derived Induced Pluripotent Stem Cells

Masoud Nasri, MSc, Benjamin Dannenmann, Perihan Mir, Malte U Ritter, Diana Amend, Yun Xu, Maksim Klimiankou, Cornelia Zeidler, Karl Welte, Julia Skokowa, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 434.
DOI: https://doi.org/10.1182/blood-2019-129991
Published: 2019
Journal Articles

Timing the Initiation of Multiple Myeloma

Francesco Maura, MD, Even H Rustad, MD, Niccolò Bolli, MD PhD, Venkata Yellapantula, PhD, Daniel A. Leongamornlert, PhD, Ferran Nadeu, BSc, MSc, Nicos Angelopoulos, PhD, Kevin J Dawson, Thomas J Mitchell, Rob Osborne, Bachisio Ziccheddu, PhD, Cristiana Carniti, PhD, Vittorio Montefusco, Paolo Corradini, Kenneth C. Anderson, MD, Philippe Moreau, MD, Elli Papaemmanuil, PhD, Ludmil Alexandrov, Xose S Puente, Elias Campo, Reiner Siebert, PhD, Herve Avet-Loiseau, MD PhD, Nikhil C. Munshi, MD, Peter J Campbell, Ola Landgren, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 573.
DOI: https://doi.org/10.1182/blood-2019-124357
Published: 2019
Journal Articles

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

Giorgia Scapin, PhD, Dhvanit I Shah, PhDPharmD
Journal: Blood
Blood (2019) 134 (Supplement_1): 445.
DOI: https://doi.org/10.1182/blood-2019-121948
Published: 2019
Journal Articles

Genetically Engineered Hematopoietic Progenitors Derived from Human Induced Pluripotent Stem Cells Achieve the Feeder-Free and Robust Production of Neutrophils with the Functional Capacity In Vivo

Masashi Miyauchi, Yusuke ITO, MD, Fumio Nakahara, MD PhD, Fumi Nakamura, MD PhD, Yuki Iwasaki, Taiki Kawagoshi, Yuki Kagoya, MD PhD, Shunya Arai, MD PhD, Mineo Kurokawa, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 720.
DOI: https://doi.org/10.1182/blood-2019-126348
Published: 2019
Journal Articles

Tropomyosin 1 Genetically Constrains in Vitro Megakaryopoiesis

Christopher Thom, MD PhD, Chintan Jobaliya, Kimberly Lorenz, PhD, Jean Ann Maguire, PhD, Alyssa Gagne, Paul Gadue, PhD, Deborah L French, PhD, Benjamin F Voight, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3612.
DOI: https://doi.org/10.1182/blood-2019-121538
Published: 2019
Journal Articles

Disease Model of Atherosclerosis Using Werner Syndrome Patient-Derived Induced Pluripotent Stem Cells Reveals Excessive Inflammatory Response Against Ox-LDL

Sudip Kumar Paul, MSc, Masamitsu Sone, PhD, Hisaya Kato, MD PhD, Yoshiro Maezawa, MD PhD, Yasuo Ouchi, PhD, Koutaro Yokote, MD PhD, Koji Eto, MD, Naoya Takayama, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3615.
DOI: https://doi.org/10.1182/blood-2019-123919
Published: 2019
Journal Articles

Eltrombopag Improves Erythroid Differentiation in a Human iPSC Model of Diamond Blackfan Anemia

Husam Qanash, MSc, Kaari Linask, Jeanette Beers, Keyvan Keyvanfar, Sara Young-Baird, Waleed Hakami, Richard H. Smith, Jizhong Zou, Andre Larochelle, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1214.
DOI: https://doi.org/10.1182/blood-2019-121822
Published: 2019
Journal Articles

Transcriptional, Protein-Level and Functional Profiling of Human Fetal Liver-Derived Hematopoietic Stem Cells at Single Cell Resolution

Kim Vanuytsel, PhD, Carlos Villacorta-Martin, MSc, Wilfredo Garcia Beltran, Taylor Matte, Alejandro Balazs, PhD, George J Murphy, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1187.
DOI: https://doi.org/10.1182/blood-2019-126857
Published: 2019
Journal Articles

A Reproducible and Simple Method to Generate Red Blood Cells from Human Pluripotent Stem Cells

Marta A. Walasek, PhD, Crystal Chau, Christian Barborini, Matthew Richardson, Stephen J. Szilvassy, Sharon A. Louis, Terry E. Thomas, Allen C. Eaves, Albertus W. Wognum
Journal: Blood
Blood (2019) 134 (Supplement_1): 1189.
DOI: https://doi.org/10.1182/blood-2019-128830
Published: 2019
Journal Articles

PML Plays a Key Role in Severe Congenital Neutropenia with Mutant ELANE Causing Neutrophil Elastase Protein Misfolding

Patricia Olofsen, MSc, Paulette van Strien, BSc, Onno Roovers, BSc, Hans De Looper, BSc, Remco Hoogenboezem, MSc, Dennis Bosch, BSc, Mehrnaz Ghazvini, PhD, Eric Bindels, PhD, Emma De Pater, PhD, Ivo Touw, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 213.
DOI: https://doi.org/10.1182/blood-2019-122423
Published: 2019
Journal Articles

FT596: Translation of First-of-Kind Multi-Antigen Targeted Off-the-Shelf CAR-NK Cell with Engineered Persistence for the Treatment of B Cell Malignancies

Jode P Goodridge, PhD, Sajid Mahmood, PhD, Huang Zhu, PhD, Svetlana Gaidarova, MS, Robert Blum, Ryan Bjordahl, PhD, Frank Cichocki, PhD, Hui-yi Chu, PhD, Greg Bonello, PhD, Tom Lee, PhD, Brian Groff, Miguel Meza, BS, Bruce Walcheck, PhD, Karl-Johan Malmberg, MD PhD, Jeffrey S. Miller, MD, Dan S Kaufman, MD PhD, Bahram Valamehr, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 301.
DOI: https://doi.org/10.1182/blood-2019-129319
Published: 2019
Journal Articles

The Inherited Platelet Disorder of RUNX1 Haploinsufficiency (Familial Platelet Disorder with Associated Myeloid Malignancy (FPDMM)) Has a Marked Deficiency of Megakaryocyte-Biased Hematopoietic Progenitor Cells: Mechanistic Studies and Drug Correction

Brian Estevez, PhD, Sara Borst, BS, Danuta Jadwiga Jarocha, PhD, Varun Sudunagunta, Michael Gonzalez, PhD, Peng Gao, Kai Tan, Deborah L French, PhD, Paul Gadue, PhD, Mortimer Poncz, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 220.
DOI: https://doi.org/10.1182/blood-2019-124851
Published: 2019
Journal Articles

FT819: Translation of Off-the-Shelf TCR-Less Trac-1XX CAR-T Cells in Support of First-of-Kind Phase I Clinical Trial

Chiawei Chang, Sjoukje Van Der Stegen, PhD, Mandal Mili, PhD, Raedun Clarke, PhD, Yi-Shin Lai, PhD, Alec Witty, PhD, Pieter Lindenbergh, Bi-Huei Yang, PhD, Mushtaq Husain, PhD, Helena Shaked, PhD, Brian Groff, Laurel Stokely, Ramzey Abujarour, PhD, Tom Lee, PhD, Hui-Yi Chu, PhD, Mochtar Pribadi, PhD, Jason ORourke, PhD, Alma Gutierrez, Isabelle Riviere, PhD, Michel Sadelain, MD PhD, Bahram Valamehr, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4434.
DOI: https://doi.org/10.1182/blood-2019-130584
Published: 2019
Journal Articles

Adamts13-Cultured Red Blood Cells to Treat Thrombotic Thrombocytopenic Purpura

Kai Wang, PhD, Khulan Batbayar, PhD, Karl Roberts, Emmanuel Olivier, PhD, Eric E. Bouhassira, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 89.
DOI: https://doi.org/10.1182/blood-2019-131929
Published: 2019