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Journal Articles

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

Blood (2021) 137 (19): 2598–2608.

https://doi.org/10.1182/blood.2020008503

Published: 2021

Includes: Supplemental data

Journal Articles

Modeling and targeting of erythroleukemia by hematopoietic genome editing

Blood (2021) 137 (12): 1628–1640.

https://doi.org/10.1182/blood.2020009103

Published: 2021

Includes: Supplemental data

Journal Articles

Loss of KLF6 Recapitulates Molecular and Functional Changes Associated with Aging in Human Hematopoietic Stem and Progenitor Cells

Alejandro Roisman, PhD, Emmalee R. Adelman, BA, Hsuan-Ting Huang, PhD, Dean Wade, Daniel Bilbao, Maria E. Figueroa, MD

Blood (2019) 134 (Supplement_1): 447.

https://doi.org/10.1182/blood-2019-130800

Published: 2019

Journal Articles

Preclinical Evaluation for Engraftment of Gene-Edited CD34+ Cells with a Sickle Cell Disease Mutation in a Rhesus Transplantation Model

Naoya Uchida, MD, Linhong Li, PhD, Tina Nassehi, Morgan Yapundich, Jackson Gamer, Claire Drysdale, Juan J. Haro-Mora, PhD, Selami Demirci, PhD, Aylin Bonifacino, MS, Allen Krouse, Nathaniel S. Linde, Cornell Allen, MS, Suk See De Ravin, MD, PhD, Robert E. Donahue, VMD, Harry L. Malech, MD, Madhusudan Peshwa, PhD, John F. Tisdale, MD

Blood (2019) 134 (Supplement_1): 609.

https://doi.org/10.1182/blood-2019-122727

Published: 2019

Journal Articles

Chromatin Structure Dynamics Preserve Genome Stability in Multiple Myeloma

Antonis Kokkalis, PhD MSc, Praveen Anand, PhD, Monica S. Nair, Johannes M. Waldschmidt, MD, Julia Frede, PhD MPhil, Tushara Vijaykumar, Amy Guillaumet-Adkins, PhD, Valeriya Dimitrova, Birgit Knoechel, MD PhD, Jens G. Lohr, MD PhD

Blood (2019) 134 (Supplement_1): 1777.

https://doi.org/10.1182/blood-2019-127495

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

Blood (2019) 134 (Supplement_1): 2066.

https://doi.org/10.1182/blood-2019-131318

Published: 2019

Journal Articles

Guided Polarization of iPSC-Derived CD4SP Helper T Cells By CRISPR/Cas9-Based Genome-Editing

Hisashi Yano, MD, Tokuyuki Shinohara, PhD, Keiko Koga, PhD, Shoichi Iriguchi, PhD, Yasuyuki Miyake, Xuewei Song, Megumi Tada, Yoshiaki Kassai, PhD, Hitoshi Kiyoi, MD PhD, Shin Kaneko, MD PhD

Blood (2019) 134 (Supplement_1): 1937.

https://doi.org/10.1182/blood-2019-122193

Published: 2019

Journal Articles

GRK6 Regulates the Hemostatic Response to Injury through Its Rate-Limiting Effects on GPCR Signaling in Platelets

Xi Chen, PhD, Shuchi Gupta, Matthew Cooper, Daniel Dehelian, Xuefei Zhao, Lawrence F. Brass, MD PhD, Ulhas P. Naik, PhD, Meghna Ulhas Naik, MS, Jeffrey Benovic, Peisong Ma, PhD

Blood (2019) 134 (Supplement_1): 3611.

https://doi.org/10.1182/blood-2019-129175

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

Blood (2019) 134 (Supplement_1): 3612.

https://doi.org/10.1182/blood-2019-121538

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

Blood (2019) 134 (Supplement_1): 4636.

https://doi.org/10.1182/blood-2019-130256

Published: 2019

Journal Articles

Heme-Regulated Inhibitor (HRI) Activates Transcription Factor ATF4 to Promote BCL11A Transcription and Fetal Hemoglobin Silencing

Peng Huang, PhD, Scott A. Peslak, MD PhD, Xianjiang Lan, PhD, Eugene Khandros, MD PhD, Malini Sharma, BS, Kunhua Qin, PhD, Cheryl A Keller, PhD, Belinda Giardine, Junwei Shi, PhD, Ross C. Hardison, PhD, Gerd A. Blobel, MD PhD

Blood (2019) 134 (Supplement_1): 814.

https://doi.org/10.1182/blood-2019-123837

Published: 2019

Journal Articles

Identification of Novel Therapeutic Targets in Atm-Deficient Lymphomas Using a Whole Genome CRISPR/CAS-9 Screen

Tatjana Stankovic, MD PhD, Nicholas Davies, PhD, Louise J Tee, Andrew D Beggs, Malcolm Taylor, PhD

Blood (2019) 134 (Supplement_1): 1504.

https://doi.org/10.1182/blood-2019-129974

Published: 2019

Journal Articles

Studying the Role of ETV6 in Megakaryopoiesis and Thrombopoiesis Using a Novel CRISPR-Cas9 Halotag Genome Editing Strategy

Marlie H Fisher, Leila J. Noetzli, PhD, Michael Callaghan, MD, Walter H. Kahr, MDPhDFRCPC, Jesse W Rowley, PhD, Jorge Di Paola, MD

Blood (2019) 134 (Supplement_1): 2468.

https://doi.org/10.1182/blood-2019-131967

Published: 2019

Journal Articles

Determining Resistance Mechanisms in BRAF- mutated Multiple Myeloma

Johannes M. Waldschmidt, MD, Jake A. Kloeber, Tushara Vijaykumar, Antonis Kokkalis, PhD MSc, Praveen Anand, PhD, Sayalee Potdar, Julia Frede, PhD MPhil, Monica S. Nair, Valeriya Dimitrova, Nikhil C. Munshi, MD, Kenneth C. Anderson, MD, Andrew J. Yee, MD, Noopur S. Raje, MD, Birgit Knoechel, MD PhD, Jens G. Lohr, MD PhD

Blood (2019) 134 (Supplement_1): 316.

https://doi.org/10.1182/blood-2019-130313

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

Blood (2019) 134 (Supplement_1): 213.

https://doi.org/10.1182/blood-2019-122423

Published: 2019

Journal Articles

Enumerating Functional HSC Clones in the Zebrafish Using Gestalt

Emily M Teets, B.S., Charles Thomas Gregory, BS, Jami L Shaffer, B.S., James S. Blachly, MD, Bradley W Blaser, MD PhD

Blood (2019) 134 (Supplement_1): 3724.

https://doi.org/10.1182/blood-2019-131503

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

Blood (2019) 134 (Supplement_1): 3744.

https://doi.org/10.1182/blood-2019-130147

Published: 2019

Journal Articles

Targeting Activated Signaling Pathways for the Treatment of IKZF1 -Deleted B Lymphoblastic Leukemia

Rohit Gupta, BA, Jason H. Rogers, MS, Xin Long, PhD, Geraldo Medrano, BS, Jaime M. Reyes, BS, Michele Redell, MD PhD, Zachary T Ball, PhD, Margaret Goodell, PhD, Rachel E. Rau, MD

Blood (2019) 134 (Supplement_1): 3789.

https://doi.org/10.1182/blood-2019-127209

Published: 2019

Journal Articles

Targeting Salt-Inducible Kinase 3 As a Therapeutic Approach for Acute Myeloid Leukemia

Shan Lin, Yusuke Tarumoto, Jinhua Wang, PhD, Joseph Milazzo, Yali Xu, Nathanael Gray, Christopher Vakoc, Kimberly Stegmaier, MD

Blood (2019) 134 (Supplement_1): 3941.

https://doi.org/10.1182/blood-2019-125278

Published: 2019

Journal Articles

Creating and Testing a CD207+ Langerhans Cell Histiocytosis-like Cell Line

Jessica Velazquez, BS, Amel Sengal, BS, Carl E. Allen, MD PhD, Rikhia Chakraborty, PhD

Blood (2019) 134 (Supplement_1): 5399.

https://doi.org/10.1182/blood-2019-130588

Published: 2019

NARROW

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

Modeling and targeting of erythroleukemia by hematopoietic genome editing

Loss of KLF6 Recapitulates Molecular and Functional Changes Associated with Aging in Human Hematopoietic Stem and Progenitor Cells

Preclinical Evaluation for Engraftment of Gene-Edited CD34+ Cells with a Sickle Cell Disease Mutation in a Rhesus Transplantation Model

Chromatin Structure Dynamics Preserve Genome Stability in Multiple Myeloma

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

Guided Polarization of iPSC-Derived CD4SP Helper T Cells By CRISPR/Cas9-Based Genome-Editing

GRK6 Regulates the Hemostatic Response to Injury through Its Rate-Limiting Effects on GPCR Signaling in Platelets

Tropomyosin 1 Genetically Constrains in Vitro Megakaryopoiesis

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

Heme-Regulated Inhibitor (HRI) Activates Transcription Factor ATF4 to Promote BCL11A Transcription and Fetal Hemoglobin Silencing

Identification of Novel Therapeutic Targets in Atm-Deficient Lymphomas Using a Whole Genome CRISPR/CAS-9 Screen

Studying the Role of ETV6 in Megakaryopoiesis and Thrombopoiesis Using a Novel CRISPR-Cas9 Halotag Genome Editing Strategy

Determining Resistance Mechanisms in BRAF- mutated Multiple Myeloma

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

Enumerating Functional HSC Clones in the Zebrafish Using Gestalt

Characterizing Dyskeratosis Congenita Caused By Parn Mutations in the Zebrafish

Targeting Activated Signaling Pathways for the Treatment of IKZF1 -Deleted B Lymphoblastic Leukemia

Targeting Salt-Inducible Kinase 3 As a Therapeutic Approach for Acute Myeloid Leukemia

Creating and Testing a CD207+ Langerhans Cell Histiocytosis-like Cell Line

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American Society of Hematology

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