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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.
https://doi.org/10.1182/blood-2019-129991
Published: 2019
Journal Articles

Mutation-Specific Dominant Negative Effects Determine the Phenotype in SRP54 D eficiency

Christoph Schuerch, MSc, Thorsten Schaefer, PhD, Joelle S. Müller, MSc, Pauline Hanns, MSc, Jonas Schärer, BSc, Julia Skokowa, MD PhD, Karl Welte, Raphael Carapito, PhD, Seiamak Bahram, MD, Martina Konantz, PhD, Claudia Lengerke, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 437.
https://doi.org/10.1182/blood-2019-127007
Published: 2019
Journal Articles

CircPCMTD1 : A Protein-Coding Circular RNA That Regulates DNA Synthesis in Leukemic Myeloblasts

Dimitrios Papaioannou, MD, Remi Buisson, Andreas Petri, PhD, Allison Walker, Marius Bill, MD PhD, Felice Pepe, PhD, Deedra Nicolet, MS, Lauren A Woodward, Krzysztof Mrózek, MD PhD, Malith Karunasiri, Gregory K. Behbehani, MD PhD, Emanuele Cocucci, PhD, Guramrit Singh, PhD, Sakari Kauppinen, PhD, Adrienne M. Dorrance, PhD, Clara D. Bloomfield, MD, Ramiro Garzon, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 640.
https://doi.org/10.1182/blood-2019-126223
Published: 2019
Journal Articles

Gene Editing ELANE in Human Hematopoietic Stem and Progenitor Cells Reveals Disease Mechanisms and Therapeutic Strategies for Severe Congenital Neutropenia

Shuquan Rao, PhD, Josias Brito-Frazao, Anna V. Serbin, Qiuming Yao, PhD, Kevin Luk, Yuxuan Wu, PhD, Jing Zeng, MD, Chunyan Ren, PhD, Ruth Watkinson, Myriam Armant, PhD, Luca Pinello, PhD, Akiko Shimamura, MD PhD, Benhur Lee, Scot A. Wolfe, PhD, Peter E. Newburger, MD, Daniel E. Bauer, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3.
https://doi.org/10.1182/blood-2019-131073
Published: 2019
Journal Articles

CRISPR-Cas9 Genome Editing of γ-Globin Promoters in Human Hematopoietic Stem Cells to Induce Erythrocyte Fetal Hemoglobin for Treatment of β-Hemoglobinopathies

Jean-Yves Metais, PhD, Phillip A Doerfler, PhD, Thiyagaraj Mayuranathan, Daniel E. Bauer, MD PhD, Stephanie Fowler, Matthew Hsieh, MD, Varun Katta, Sagar Keriwala, Cicera Lazzarotto, Kevin Luk, Michael Neel, Scott Perry, Shaina Porter, Byoung Y. Ryu, PhD, Akshay Sharma, MBBS, Devlin Shea, Samuel Peters, John F. Tisdale, MD, Scot A. Wolfe, PhD, Kaitly Jensen Woodard, BS, Naoya Uchida, MD, Yuxuan Wu, PhD, Yu Yao, MD, Jing Zeng, MD, Shondra M. Pruett-Miller, PhD, Shengdar Q. Tsai, PhD, Mitchell J. Weiss, MD, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2066.
https://doi.org/10.1182/blood-2019-131318
Published: 2019
Journal Articles

Combining CAR Engineering and CIS Checkpoint Deletion in NK Cells for the Treatment of B Cell Hematologic Malignancies

May Daher, MD, Rafet Basar, MD, Elif Gokdemir, BS, Natalia Baran, PhDMD, Nadima Uprety, MS, Mayela Carolina Mendt, PhD, Lucila Kerbauy, MD, Mayra Shanley, PhD, Ana Karen Nunez Cortes, MD, Li Li, MD, Pinaki P Banerjee, PhD, Vakul Mohanty, PhD, Junjun Lu, Gonca Ozcan, MD, Emily Ensley, BS, Vandana Nandivada, BS, MS, Mecit Kaplan, MS, Mustafa H Bdaiwi, MD, Enli Liu, MD, Sonny Ang, PhD, Ken Chen, PhD, Marina Y Konopleva, MD PhD, Richard E. Champlin, MD, Elizabeth J. Shpall, MD, Katayoun Rezvani, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1936.
https://doi.org/10.1182/blood-2019-126739
Published: 2019
Journal Articles

EDIT-301: An Experimental Autologous Cell Therapy Comprising Cas12a-RNP Modified mPB-CD34+ Cells for the Potential Treatment of SCD

Edouard De Dreuzy, PhD, Jack Heath, John A Zuris, PhD, Patricia Sousa, Ramya Viswanathan, Sean Scott, Jen Da Silva, Terence Ta, PhD, Stacy Capehart, PhD, Tongyao Wang, Cecilia Fernandez, PhD, Vic E Myer, PhD, Charles F Albright, PhD, Christopher J Wilson, PhD, Sandra Teixeira, PhD, Kai-Hsin Chang, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4636.
https://doi.org/10.1182/blood-2019-130256
Published: 2019
Journal Articles

Invariant NKT Cells Recognize Leukemia Cells with T-Cell and NK Receptors in a CD1d-Independent Manner

Takahiro Aoki, MD, Mariko Takami, Tomozumi Takatani, Kiwamu Motoyoshi, Ayana Ishii, Ayaka Hara, Takahide Toyoda, Reona Okada, Moeko Hino, MD, Ryo Koyama-Nasu, Masahiro Kiuchi, Kiyoshi Hirahara, Toshinori Nakayama, Naoki Shimojo, Shinichiro Motohashi
Journal: Blood
Blood (2019) 134 (Supplement_1): 3225.
https://doi.org/10.1182/blood-2019-121817
Published: 2019
Journal Articles

FLT3-ITD Activates Cytoplasmic Drosha-Dependent Non-Canonical Mechanisms of Mir-155 Biogenesis in Acute Myeloid Leukemia

Le Xuan Truong Nguyen, PhD, Bin Zhang, PhD, Dinh Hoa Hoang, BS, Dandan Zhao, MD, Sergio Branciamore, Davide Maestrini, PhD, Yu-Lin SU, PhD, Sonia Rodriguez, PhD, Flavia Pichiorri, PhD, Steven Rosen, MD, Michael A. Caligiuri, MD, Stephen J. Forman, MD, Ling Li, PhD, Marcin Kortylewski, PhD, Russell Rockne, PhD, Ya-Huei Kuo, PhD, Nadia Carlesso, MD, Guido Marcucci, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2722.
https://doi.org/10.1182/blood-2019-131871
Published: 2019
Journal Articles

Interactome Analysis of APOBEC3B in Multiple Myeloma

Yasuhiro Kazuma, MD, Kotaro Shirakawa, MD PhD, Anamaria Daniela Sarca, MD, Yoshihito Horisawa, MD, Hirofumi Fukuda, MD, Hiroyuki Matsui, MD, Hiroyuki Yamazaki, MD, Tadahiko Matsumoto, MD, Fumiko Tanabe, Ryo Morishita, PhD, Shinji Ito, PhD, Akifumi Takaori-Kondo, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1259.
https://doi.org/10.1182/blood-2019-126856
Published: 2019
Journal Articles

A Novel Source of Endogenous DNA Damage That Requires Repair By the Fanconi Anemia Pathway

Moonjung Jung, MD, Sunandini Sridhar, PhD, Audrey Goldfarb, BA, Ryan White, PhD, Danielle Keahi, BA, Raymond Noonan, BA, Tom Wiley, BS, Francis P. Lach, BS, Agata Smogorzewska, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 106.
https://doi.org/10.1182/blood-2019-127686
Published: 2019
Journal Articles

Identification of ST3GAL6-AS1 Interactive Protein in Multiple Myeloma

Ying Shen, MD, Fangmei Li, PhD, Dong Wu, MD, Yue Peng, Wanhong Zhao, MD, Yun Yang, MD, Jianli Wang, MD, Ju Bai, PhD, Yan Xu, Aili He
Journal: Blood
Blood (2019) 134 (Supplement_1): 4365.
https://doi.org/10.1182/blood-2019-125210
Published: 2019
Journal Articles

Characterizing Dyskeratosis Congenita Caused By Parn Mutations in the Zebrafish

Anna Cordeiro, PhD, Adam P Deveau, MSc, Santhosh Dhanraj, PhD, Yigal Dror, MDFRCPC, Jason Berman, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3744.
https://doi.org/10.1182/blood-2019-130147
Published: 2019
Journal Articles

A Functional Analysis of SNP on Anthracycline-Induced Cardiotoxicity in a Uniform Genetic Background Using CRISPR/Cas9 Mediated Single-Base-Edited iPSCs

Takayuki Nakamura, MD, Shinichi Mizuno, MD PhD, Hidetoshi Ozawa, Yoshitaka Yamasaki, MD, Shuki Oya, MD, Satoshi Morishige, MD, Kazutoshi Aoyama, MD PhD, Maki Ymaguchi, MD PhD, Ritsuko Seki, PhD, Fumihiko Mouri, MD PhD, Koichi Osaki, MD, Koji Nagafuji, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 5748.
https://doi.org/10.1182/blood-2019-123932
Published: 2019
Journal Articles

Role of Mutant p53 in the Progression of Chronic Lymphocytic Leukemia

Janani Ravikrishnan, Tzung-Huei Lai, PhD DVM, Elizabeth Muhowski, Lindsey Brinton, PhD, Katie Williams, MS, Alexandria Thompson, James S. Blachly, MD, Hatice Gulcin Ozer, PhD, John C. Byrd, MD, Rosa Lapalombella, PhD, Jennifer Woyach, MD, Deepa Sampath, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2526.
https://doi.org/10.1182/blood-2019-128574
Published: 2019
Journal Articles

Recreation of t(7;12)(q36;p13) in Human Induced Pluripotent Stem Cells Using CRISPR/Cas9 Generates a Useful Model for Studying Acute Myeloid Leukemia

Tina Nilsson, MSc, BSc, Ahmed Waraky, PhD MSc, BSc, Anders Östlund, MSc, BSc, Laleh Arabanian, PhD, Julia Asp, PhD, Linda Fogelstrand, Lars Palmqvist, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2736.
https://doi.org/10.1182/blood-2019-123004
Published: 2019
Journal Articles

Imetelstat Sensitizes Hematopoietic Malignancy Cells to Genotoxic Agent Via Suppression of Telomerase Mediated DNA Repair Process

Daisuke Hidaka, Masahiro Onozawa, Naohiro Miyashita, Masao Nakagawa, Daigo Hashimoto, Takanori Teshima, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3369.
https://doi.org/10.1182/blood-2019-122999
Published: 2019
Journal Articles

Interrogating RNA Binding Proteins as Novel Regulators of Fetal Hemoglobin Expression

Aoi Wakabayashi, BS, Jeremy D. Grevet, PhD, Xianjiang Lan, PhD, Malini Sharma, BS, Junwei Shi, PhD, Gerd A. Blobel, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 966.
https://doi.org/10.1182/blood-2019-125901
Published: 2019
Journal Articles

Knockdown of HNF4A Gene Increases Fetal Hemoglobin Synthesis in Hudep-2

Ingrid Grazielle Sousa, BSc, MSc, Priscila Keiko Matsumoto Martin, PhD, Dulcinéia Martins de Albuquerque, BSc, PhD, Carolina Lanaro, BSc, PhD, Ryo Kurita, PhD, Yukio Nakamura, MD PhD, Fernando Ferreira Costa, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 968.
https://doi.org/10.1182/blood-2019-130920
Published: 2019
Journal Articles

Durable and Robust Fetal Globin Induction without Anemia in Rhesus Monkeys Following Autologous Hematopoietic Stem Cell Transplant with BCL11A Erythroid Enhancer Editing

Selami Demirci, PhD, Jing Zeng, MD, Yuxuan Wu, PhD, Naoya Uchida, MD, Jackson Gamer, Morgan Yapundich, Claire Drysdale, Aylin C. Bonifacino, Allen E. Krouse, Nathaniel S. Linde, Robert E. Donahue, VMD, Anne H. Shen, Juan J. Haro-Mora, PhD, Alexis Leonard, MD, Tina Nassehi, Kevin Luk, Scot A. Wolfe, Cicera R. Lazzarotto, Shengdar Q. Tsai, PhD, Mitchell J. Weiss, MD, PhD, Daniel E. Bauer, MD PhD, John F. Tisdale, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4632.
https://doi.org/10.1182/blood-2019-129394
Published: 2019