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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

Identification of Novel Variants Associated with Fetal Hemoglobin Levels in Healthy Donors (the INTERVAL study)

Emily Kawabata, Samuel Lessard, PhD, Dirk Paul, Paola G Bronson, Robert Peters, Sriram Krishnamoorthy, PhD, William Astle, Adam Butterworth

Blood (2019) 134 (Supplement_1): 2243.

https://doi.org/10.1182/blood-2019-123977

Published: 2019

Journal Articles

Combining HPFH Mutations in Human Adult HSCs to Enhance Reactivation of Fetal Hemoglobin

Nikoletta Psatha, Afrodite Georgakopoulou, PhDstudent, Chang Li, PhD, Pavel Sova, Hongjie Wang, PhD, Andre Lieber, MD PhD, Thalia Papayannopoulou, MD DrSci

Blood (2019) 134 (Supplement_1): 2246.

https://doi.org/10.1182/blood-2019-131487

Published: 2019

Journal Articles

Functional Overlap between the SEC23 Paralogs Suggests a Novel Treatment Paradigm for Congenital Dyserythropoietic Anemia Type II

Richard King, MD, Ann Friedman, BS, Zesen Lin, BS, Rami Khoriaty, MD

Blood (2019) 134 (Supplement_1): 2221.

https://doi.org/10.1182/blood-2019-129669

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

Blood (2019) 134 (Supplement_1): 4632.

https://doi.org/10.1182/blood-2019-129394

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

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

Understanding Pre-Leukemia in Trisomy 21 Human HSC and Modeling Progression Towards Down Syndrome Associated Leukemia Using CRISPR/Cas9 at Single Cell Resolution

Elvin Wagenblast, PhD, Olga I. Gan, PhD, Maria Azkanaz, Sabrina A. Smith, Joana Araújo, MD, Lorien Shakib, Jessica L. McLeod, Gabriela Krivdova, Johann K. Hitzler, MD, Eric R. Lechman, PhD, John E. Dick, PhD

Blood (2019) 134 (Supplement_1): 2531.

https://doi.org/10.1182/blood-2019-129259

Published: 2019

Journal Articles

Genome-Scale CRISPR-Cas9 Synthetic Lethal Screening of AML Cell Line Identified Functional Modulators of Etoposide Resistance Predictive of Clinical Outcome in AML Patients

Roya Rafiee, M.Sc., PhD, Amin Sobh, PhD, Abdelrahman H. Elsayed, MSc, Abderrahmane Tagmount, PhD, Chris D Vulpe, MD PhD, Jatinder K Lamba, M.Sc., PhD

Blood (2019) 134 (Supplement_1): 2685.

https://doi.org/10.1182/blood-2019-131312

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

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

Universal' Fratricide-Resistant CAR T Cells Against T Cell Leukemia Generated By Coupled & Uncoupled Deamination Mediated Base Editing

Christos Georgiadis, PhD, Jane Rasaiyaah, PhD, Soragia Athina Gkazi, PhD, Roland Preece, Annie Etuk, PhD, Abraham Christi, Waseem Qasim, MBBS, PhD

Blood (2019) 134 (Supplement_1): 3219.

https://doi.org/10.1182/blood-2019-130057

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

NARROW

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

Modeling and targeting of erythroleukemia by hematopoietic genome editing

Identification of Novel Variants Associated with Fetal Hemoglobin Levels in Healthy Donors (the INTERVAL study)

Combining HPFH Mutations in Human Adult HSCs to Enhance Reactivation of Fetal Hemoglobin

Functional Overlap between the SEC23 Paralogs Suggests a Novel Treatment Paradigm for Congenital Dyserythropoietic Anemia Type II

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

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

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

Understanding Pre-Leukemia in Trisomy 21 Human HSC and Modeling Progression Towards Down Syndrome Associated Leukemia Using CRISPR/Cas9 at Single Cell Resolution

Genome-Scale CRISPR-Cas9 Synthetic Lethal Screening of AML Cell Line Identified Functional Modulators of Etoposide Resistance Predictive of Clinical Outcome in AML Patients

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

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

Universal' Fratricide-Resistant CAR T Cells Against T Cell Leukemia Generated By Coupled & Uncoupled Deamination Mediated Base Editing

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

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American Society of Hematology

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American Society of Hematology

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