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Journal Articles

Ex vivo Engineering of Red Blood Cells

Jan Frayne, BsC, PhD, Deborah E Daniels, Jan Frayne, BsC PhD, Timothy J Satchwell, PhD, Joseph Hawksworth, BSc PhD, Belinda K Singleton, Tatyana N Andrienko, PhD, Marieangela C Wilson, Sabine Kupzig, Marjolein Meinders, Ryo Kurita, PhD, Yukio Nakamura, MD PhD, Ivan Ferrer Vicens, PhD MSc, BSc, Rebecca Griffiths, PhD, Nicola Cogan, Kongtana Trakarnsanga, MD PhD, David J. Anstee, PhD FRCPath, Ashley Mark Toye, PhD

Blood (2019) 134 (Supplement_1): SCI-5.

https://doi.org/10.1182/blood-2019-121117

Published: 2019

Journal Articles

CRISPR/Cas9-mediated in vivo gene targeting corrects hemostasis in newborn and adult factor IX–knockout mice

Lili Wang, Yang Yang, Camilo Ayala Breton, John White, Jia Zhang, Yan Che, Alexei Saveliev, Deirdre McMenamin, Zhenning He, Caitlin Latshaw, Mingyao Li, James M. Wilson

Blood (2019) 133 (26): 2745–2752.

https://doi.org/10.1182/blood.2019000790

Published: 2019

Includes: Supplemental data

Journal Articles

MMP9 Inhibition Rescues the Erythroid Defect in RPS14-Deficient Del(5q) MDS Models

Minyoung Youn, PhD, Haigen Huang, PhD, Cheng Chen, Sharon Kam, BS, Mark C Wilkes, Hee-Don Chae, PhD, Anupama Narla, MD, Shuo Lin, PhD, Kathleen M. Sakamoto, MD PhD

Blood (2018) 132 (Supplement 1): 756.

https://doi.org/10.1182/blood-2018-99-117508

Published: 2018

Journal Articles

The enigma of monosomy 7

Toshiya Inaba, Hiroaki Honda, Hirotaka Matsui

Blood (2018) 131 (26): 2891–2898.

https://doi.org/10.1182/blood-2017-12-822262

Published: 2018

Journal Articles

Functional Analyses of BTK Mutations in Follicular Lymphoma

Nan Hu, PhD, Fangyang Wang, Denzil Bernard, Shaomeng Wang, Mark Kaminski, Sumana Devata, MD, Tycel J. Phillips, MD, Sami N. Malek, MD

Blood (2017) 130 (Supplement 1): 647.

https://doi.org/10.1182/blood.V130.Suppl_1.647.647

Published: 2017

Journal Articles

Preclinical Development of HBB Gene Correction in Autologous Hematopoietic Stem and Progenitor Cells to Treat Severe Sickle Cell Disease

Daniel P. Dever, PhD, Joab Camarena, BS, Ciaran Lee, PhD, Christopher Vakulskas, PhD, Mark Behlke, MD PhD, Gang Bao, PhD, Matthew Porteus, MD PhD

Blood (2017) 130 (Supplement 1): 4620.

https://doi.org/10.1182/blood.V130.Suppl_1.4620.4620

Published: 2017

Journal Articles

A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta-Thalassemia and Sickle Cell Disease

Hao Bai, PhD, Liuhong Cai, MD, Vasiliki Machairaki, PhD, Yongxing Gao, Zhaohui Ye, PhD, Linzhao Cheng, PhD

Blood (2017) 130 (Supplement 1): 5564.

https://doi.org/10.1182/blood.V130.Suppl_1.5564.5564

Published: 2017

Journal Articles

A Missense Mutation in the Loss-of-Function Intolerant Actr2 Gene Suppresses Thrombosis Via Multiple Mechanisms

Amy E Siebert, MS, Linzi M Hobbs, Alexander J Johnston, Lena M Mishack, Marisa A Brake, BS, Audrey Cleuren, PhD, Guojing Zhu, MD, Randal Joseph Westrick, PhD

Blood (2017) 130 (Supplement 1): 1058.

https://doi.org/10.1182/blood.V130.Suppl_1.1058.1058

Published: 2017

Journal Articles

Wt1 Haploinsufficiency Induces an Age-Dependent Enhancement in Stem Cell Self-Renewal and Cooperates with FLT3-ITD to Drives Leukemogenesis

Elodie Pronier, PhD, Justin T Whitfield, Tiffany Merlinsky, Jihae Ahn, Raajit K. Rampal, MD PhD, Benjamin H Durham, MD BS, Ari Melnick, MD, Ross L. Levine, MD

Blood (2017) 130 (Supplement 1): 1214.

https://doi.org/10.1182/blood.V130.Suppl_1.1214.1214

Published: 2017

Journal Articles

Crispr/Cas9-Mediated In Vivo Gene Targeting Corrects Haemostasis in Newborn and Adult FIX-KO Mice

Lili Wang, PhD, Yang Yang, PhD, John White, McMenamin Deirdre, Peter Bell, PhD, James M Wilson, MD PhD

Blood (2016) 128 (22): 1174.

https://doi.org/10.1182/blood.V128.22.1174.1174

Published: 2016

Journal Articles

Long-Term Expression of Von Willebrand Factor Via Piggybac -Mediated Gene Transfer

Blake A Johnson, Anil K. Chauhan, PhD, Janice M Staber, MD

Blood (2016) 128 (22): 3511.

https://doi.org/10.1182/blood.V128.22.3511.3511

Published: 2016

Journal Articles

Beta-Globin Gene Targeting in Human Hematopoietic Stem Cells Using Cas9 Ribonucleoprotein and rAAV6

Daniel Patrick Dever, PhD, Matthew Porteus, MD PhD

Blood (2016) 128 (22): 2310.

https://doi.org/10.1182/blood.V128.22.2310.2310

Published: 2016

Journal Articles

Genome Editing of Factor V in Zebrafish Embryos Results in a Severe Hemostatic Defect without Spontaneous Hemorrhage

Angela C Weyand, MD, Steve J Grzegorski, Jordan A. Shavit, MD PhD

Blood (2016) 128 (22): 2565.

https://doi.org/10.1182/blood.V128.22.2565.2565

Published: 2016

Journal Articles

FANCN Hypomorphic Mutation Retains BRCA1 Binding Domain

Elizabeth Thompson, MS, Jakub Tolar, MD PhD, Eric A. Hendrickson, PhD

Blood (2016) 128 (22): 2676.

https://doi.org/10.1182/blood.V128.22.2676.2676

Published: 2016

Journal Articles

Genome-Scale ORF Screen for Mediators of NF-κb Activation in DLBCL

Hee Min Yoo, PhD, Roland Schmitz, Xin Yu, Da Wei Huang, James D. Phelan, Daniel E. Webster, Louis M. Staudt, MD PhD

Blood (2016) 128 (22): 4102.

https://doi.org/10.1182/blood.V128.22.4102.4102

Published: 2016

Journal Articles

Welcoming a new age for gene therapy in hematology

Mitchell J. Weiss, Charles G. Mullighan

Blood (2016) 127 (21): 2523–2524.

https://doi.org/10.1182/blood-2016-03-678714

Published: 2016

Journal Articles

Engineering the Cancer Genome

Tyler Jacks, PhD

Blood (2015) 126 (23): SCI-19.

https://doi.org/10.1182/blood.V126.23.SCI-19.SCI-19

Published: 2015

Journal Articles

Platelet Phosphatidylserine Exposure, Survival and Blood Coagulation in Mice Lacking TMEM16F

Gary E. Gilbert, MD, Colin Kretz, PhD, David Ginsburg, MD, Valerie Anne Novakovic, MS

Blood (2015) 126 (23): SCI-32.

https://doi.org/10.1182/blood.V126.23.SCI-32.SCI-32

Published: 2015

Journal Articles

Allele-Specific Crispr Targeting Reveals Epigenetic and Phenotypic Effects of a MMSET Gain of Function Mutation Found in Relapsed Acute Lymphoblastic Leukemia

Jon Oyer, PhD, Christine Will, M.S., Xiaoxiao Huang, Teresa Ezponda, PhD, Relja Popovic, PhD, Yupeng Zheng, PhD, Neil L Kelleher, PhD, Jonathan D. Licht, MD

Blood (2015) 126 (23): 437.

https://doi.org/10.1182/blood.V126.23.437.437

Published: 2015

Journal Articles

Bleeding Phenotype Observed in Murine Defect of VWF-Collagen 4 Interactions

Veronica H Flood, MD, Tricia L Slobodianuk, Ashley Doruelo, Derek Adams, Jamie Foeckler, Shawn Kalloway, Hartmut Weiler, Robert R Montgomery

Blood (2015) 126 (23): 211.

https://doi.org/10.1182/blood.V126.23.211.211

Published: 2015

NARROW

Ex vivo Engineering of Red Blood Cells

CRISPR/Cas9-mediated in vivo gene targeting corrects hemostasis in newborn and adult factor IX–knockout mice

MMP9 Inhibition Rescues the Erythroid Defect in RPS14-Deficient Del(5q) MDS Models

The enigma of monosomy 7

Functional Analyses of BTK Mutations in Follicular Lymphoma

Preclinical Development of HBB Gene Correction in Autologous Hematopoietic Stem and Progenitor Cells to Treat Severe Sickle Cell Disease

A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta-Thalassemia and Sickle Cell Disease

A Missense Mutation in the Loss-of-Function Intolerant Actr2 Gene Suppresses Thrombosis Via Multiple Mechanisms

Wt1 Haploinsufficiency Induces an Age-Dependent Enhancement in Stem Cell Self-Renewal and Cooperates with FLT3-ITD to Drives Leukemogenesis

Crispr/Cas9-Mediated In Vivo Gene Targeting Corrects Haemostasis in Newborn and Adult FIX-KO Mice

Long-Term Expression of Von Willebrand Factor Via Piggybac -Mediated Gene Transfer

Beta-Globin Gene Targeting in Human Hematopoietic Stem Cells Using Cas9 Ribonucleoprotein and rAAV6

Genome Editing of Factor V in Zebrafish Embryos Results in a Severe Hemostatic Defect without Spontaneous Hemorrhage

FANCN Hypomorphic Mutation Retains BRCA1 Binding Domain

Genome-Scale ORF Screen for Mediators of NF-κb Activation in DLBCL

Welcoming a new age for gene therapy in hematology

Engineering the Cancer Genome

Platelet Phosphatidylserine Exposure, Survival and Blood Coagulation in Mice Lacking TMEM16F

Allele-Specific Crispr Targeting Reveals Epigenetic and Phenotypic Effects of a MMSET Gain of Function Mutation Found in Relapsed Acute Lymphoblastic Leukemia

Bleeding Phenotype Observed in Murine Defect of VWF-Collagen 4 Interactions

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American Society of Hematology

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American Society of Hematology

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