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Journal Articles

Informing Clinical Decision and Policy Making in Blood Related Disorders Using Targeted Next Generation Sequencing

Betoul Baz, PhD, Tarek Owaidah, MDFRCPA, Majed Dasouki, MD, Mohammed Abouelhoda, PhD, Dorota Monies, PhD, Nada Al Tassan, PhD

Blood (2019) 134 (Supplement_1): 5776.

https://doi.org/10.1182/blood-2019-122370

Published: 2019

Journal Articles

Characterization of Pathogenic Variants Associated with Hereditary Thrombocytopenias in Families from the Czech Republic

Zuzana Vrzalova, PhD, Katerina Stano Kozubik, PhD, Lenka Radova, PhD, Jakub Trizuljak, MD, Sarka Pospisilova, Prof PhD, Michael Doubek, MD PhD

Blood (2019) 134 (Supplement_1): 2343.

https://doi.org/10.1182/blood-2019-122696

Published: 2019

Journal Articles

A Prospective Phase II, Open-Label, Single-Arm, Multicenter Study to Assess the Efficacy and Safety of SEG101 (Crizanlizumab) in Sickle Cell Disease Patients with Priapism (SPARTAN)

Fuad A El Rassi, MD, Deepika S. Darbari, MD, Arthur Burnett, MD MBA, Jincy Paulose, MD, Dramane I Laine, Das Purkayastha, PhD, Gregory J Kato, MD

Blood (2019) 134 (Supplement_1): 1007.

https://doi.org/10.1182/blood-2019-125790

Published: 2019

Journal Articles

Development of a Personalized 3D Carotid Model for Cerebral Vasculopathy Monitoring in Sickle Cell Disease

Nguyen-Peyre Kim-Anh, PhD, Laurène Lenoir, Valentin Amar, Frédéric Segonds, Nuriye Akçay, Seguin Marion, Philippe Connes, PhD, France Pirenne, MD PhD, Jean-Frédéric Gerbeau, Irène Vignon-Clémentel, Suzanne Verlhac, MD, Pablo Bartolucci, MD PhD

Blood (2019) 134 (Supplement_1): 3570.

https://doi.org/10.1182/blood-2019-124327

Published: 2019

Journal Articles

NHEJ-Mediated Gene Editing, a Versatile Approach to Correct a Variety of Fanconi Anemia Genes in HSCs

Francisco J Roman-Rodriguez, PhD, Laura Ugalde, Lara A. Alvarez, Begoña Díez-Cabezas, PhD, Jordi Surrallés, PhD, Maria Jose Ramirez, PhD, Massimo Bogliolo, PhD, Helmut Hanenberg, MD PhD, Julian Sevilla, MD PhD, Sandra Rodriguez-Perales, PhD, Raul Torres-Ruiz, PhD, Juan A. Bueren, PhD, Paula Rio, PhD

Blood (2019) 134 (Supplement_1): 4639.

https://doi.org/10.1182/blood-2019-129946

Published: 2019

Journal Articles

Liver-Directed AAV Gene Therapy for Gaucher Disease

Carlos J Miranda, PhD, Miriam Canavese, PhD, Elisa Chisari, MS, Jalpa Pandya, MPhil, Clement Cocita, PhD, Maria Portillo, MRes, Jenny McIntosh, PhD, Azadeh Kia, PhD, Jonathan H. Foley, PhD, Allison Dane, PhD, Jey M Jeyakumar, PhD, Rose Sheridan, PhD, Romuald Corbau, Amit C. Nathwani, MD PhD

Blood (2019) 134 (Supplement_1): 3354.

https://doi.org/10.1182/blood-2019-124280

Published: 2019

Journal Articles

The Rare Diseases Pilot for the 100,000 Genomes Project: Findings in Known and New Genes by Analysis of 3,549 Whole Genome Sequenced Samples from Patients and Relatives with Haematological, Haemostasis and Immune Disorders

Suthesh Sivapalaratnam, MD PhD, Nihr Bioresource

Blood (2018) 132 (Supplement 1): 504.

https://doi.org/10.1182/blood-2018-99-119068

Published: 2018

Journal Articles

Persistence of CRISPR/Cas9-Edited Hematopoietic Stem and Progenitor Cells and Reactivation of Fetal Hemoglobin in Nonhuman Primates

Olivier Humbert, PhD, Stefan Radtke, PhD, Ray R Carillo, Anai M Perez, Sowmya Somashekar Reddy, Lauren E Schefter, Christopher Lux, MDPhD, Sowmya Pattabhi, PhD, Olivier Negre, PhD, Ciaran M Lee, PhD, Jennifer Adair, PhD, Christopher Peterson, PhD, Gang Bao, PhD, David J. Rawlings, MD, Andrew M. Scharenberg, MD, Hans-Peter Kiem, MD PhD

Blood (2018) 132 (Supplement 1): 806.

https://doi.org/10.1182/blood-2018-99-112996

Published: 2018

Journal Articles

Potential Long-Term Treatment of Hemophilia a By Early Postnatal Co-Transplantation of Cord Blood Derived Endothelial Colony-Forming Cells and Placental Mesenchymal Stem Cells

Kewa Gao, MD, Priyadarsini Kumar, PhD, Dake Hao, PhD, Lizette Reynaga, BS, Diana Lee Farmer, MD, Melanie Rose, MS, Jan A. Nolta, PhD, Jianda Zhou, MD PhD, Ping Zhou, PhD, Aijun Wang, PhD

Blood (2018) 132 (Supplement 1): 3318.

https://doi.org/10.1182/blood-2018-99-117701

Published: 2018

Journal Articles

Precision Medicine for Sickle Cell Disease through Whole Genome Sequencing

Blood (2018) 132 (Supplement 1): 3641.

https://doi.org/10.1182/blood-2018-99-117606

Published: 2018

Journal Articles

A Novel Filtering Tool for Detecting Sickle Cell Disease in the Pediatric Population

Evashin Pillay, MD, Theresa Louise Coetzer, PhD

Blood (2018) 132 (Supplement 1): 2238.

https://doi.org/10.1182/blood-2018-99-119121

Published: 2018

Journal Articles

Gene Therapy for Thalassemia and Sickle Cell Anemia Poses Acceptable Toxicity and Efficacy: Clinical Outcomes in Forty-Seven Cases

Hafiz Muhammad Fazeel, MD, Muhammad Abu Zar, MD, Saad Ullah Malik, MD, Ahmad Kamal, MD, Mustafa Nadeem Malik, MD, Syed Shabee Hassan, MD, Syeda Sabeeka Batool, MD, Ceren Durer, MD, Seren Durer, MD, Shehroz Aslam, MD, Madeeha Shafqat, Faiqa Ijaz, MBBS, Faiz Anwer, MD, Shahrukh K. Hashmi, MDMPH

Blood (2018) 132 (Supplement 1): 2194.

https://doi.org/10.1182/blood-2018-99-114511

Published: 2018

Journal Articles

Non-Myeloablative Transplant in Severe Sickle Cell Disease Is Safe & Effective. Single Center Experience from Saudi Arabia

Mohsen Al-Zahrani, MD, Ahmed Alaskar, MD FRCPC, Moussab Damlaj, MD, Khadega Abuelgasim, MD, Giamal Gmati, MD, Hind ABDIN Salama, MD, Samer Ghazi, Osama Ali, MD, Bader Alahmari, MD, Ayman Hejazi, MD

Blood (2017) 130 (Supplement 1): 5549.

https://doi.org/10.1182/blood.V130.Suppl_1.5549.5549

Published: 2017

Journal Articles

Talen Mediated Therapeutic Gene Editing Strategy for ß-Hemoglobinopathies

Christopher Lux, MDPhD, Kyle Jacoby, PhD, Sowmya Pattabhi, PhD, Julia Yang, BS, Olivier Humbert, PhD, Brian Gordon, MS, Irwin D. Bernstein, MD, David J. Rawlings, MD, Andrew M. Scharenberg, MD

Blood (2017) 130 (Supplement 1): 3340.

https://doi.org/10.1182/blood.V130.Suppl_1.3340.3340

Published: 2017

Journal Articles

High Prevalence of Severe Acute Malnutrition in Children with Sickle Cell Anemia over 5 Years of Age in Northern Nigeria

Djamila Labib Ghafuri, MD, Shehu Umar Abdullahi, Najibah Aliyu Galadanci, MBBS,MPH, Binta Jibir, MBBS MPH, Safiya Gambo, MBBS, Halima Bello-Manga, MBBS, MPH, Lawal Haliru, MBBS, Khadija Bulama, BNSc, Awwal Gambo, Muktar Aliyu, MBBS DrPH MPH, Brittany Covert, MPH, CCRP, Adetola A. Kassim, MD MS, Mark Rodeghier, PhD, Michael R. DeBaun, MD MPH

Blood (2017) 130 (Supplement 1): 127.

https://doi.org/10.1182/blood.V130.Suppl_1.127.127

Published: 2017

Journal Articles

Reprogramming Cell-Signaling By Delivering the Catalytic Domain of Ptpgr Ameliorates Anemia of β-Thalassemia

Alessandro Mattè, PhD, Elisabetta Beneduce, Michela Mirenda, PhD, Roberta Russo, Achille Iolascon, MD PhD, Antonella Pantaleo, PhD, Francesco Turrini, MD PhD, Angela Siciliano, Enrica Federti, PhD, Serge Cedrick, Anne Janin, PhD, Christophe Leboeuf, Carlo Laudanna, MD, Lucia De Franceschi, MD

Blood (2017) 130 (Supplement 1): 2219.

https://doi.org/10.1182/blood.V130.Suppl_1.2219.2219

Published: 2017

Journal Articles

The NLRP3 Inflammasome in Platelets Is Upregulated in Sickle Cell Disease and Promotes Platelet Aggregation and In Vitro Thrombosis

Sebastian Vogel, MD PhD, Taruna Arora, MD, Xunde Wang, PhD, Luis E. Almeida, MD PhD, Zenaide Quezado, MD, Swee Lay Thein, DSc,FRCP,FRCPath,MBBS,MRCPath,MRCP

Blood (2017) 130 (Supplement 1): 2234.

https://doi.org/10.1182/blood.V130.Suppl_1.2234.2234

Published: 2017

Journal Articles

Novel Lineage Depletion and Manufacturing Allows for Unprecedented Preservation of Autologous Blood Stem Cells for Gene Therapy of Fanconi Anemia Complementation Group Α

Jennifer Adair, PhD, Devikha Chandrasekaran, MS, Stefanie Schmuck, BS, Gabriella Sghia-Hughes, BS, Grace Choi, BS, Anne Lee, BS, Kevin G Haworth, BS, PhD, Ann Woolfrey, MD, Lauri S Burroughs, MD, Pamela S. Becker, MD PhD, Hans-Peter Kiem, MD PhD

Blood (2017) 130 (Supplement 1): 2451.

https://doi.org/10.1182/blood.V130.Suppl_1.2451.2451

Published: 2017

Journal Articles

TET2 and MSH6: A Novel Role for MMR in the Pathogenesis of Myeloid Malignancies

Edward F Greenberg, MD, Cassandra M. Hirsch, BS, Feng Pan, PhD, Babal K Jha, PhD, Yihong Guan, PhD, Valeria Visconte, PhD, Dewen You, PhD, Guangbo Qu, PhD, Amy Graham, Bartlomiej P. Przychodzen, MSc, Thomas Laframboise, PhD, Hideki Makishima, MD PhD, Daniel Gackowski, PhD, Ryszard Ólinski, Professor, Yu-Wei Cheng, PhD, Mikkael A. Sekeres, MD MS, Mingjiang Xu, MDPhD, Jaroslaw P. Maciejewski, PhD MD FACP

Blood (2017) 130 (Supplement 1): 1668.

https://doi.org/10.1182/blood.V130.Suppl_1.1668.1668

Published: 2017

Journal Articles

Altered Blood Gene Transcription in Chuvash Polycythemia and Its Cell Lineage Specificity

Xu Zhang, PhD, Jihyun Song, PhD, Binal N. Shah, PhD, Galina Miasnikova, MD, Adelina Sergeyeva, MD, Victor R. Gordeuk, MD, Josef T. Prchal, MD

Blood (2016) 128 (22): 1244.

https://doi.org/10.1182/blood.V128.22.1244.1244

Published: 2016

NARROW

Informing Clinical Decision and Policy Making in Blood Related Disorders Using Targeted Next Generation Sequencing

Characterization of Pathogenic Variants Associated with Hereditary Thrombocytopenias in Families from the Czech Republic

A Prospective Phase II, Open-Label, Single-Arm, Multicenter Study to Assess the Efficacy and Safety of SEG101 (Crizanlizumab) in Sickle Cell Disease Patients with Priapism (SPARTAN)

Development of a Personalized 3D Carotid Model for Cerebral Vasculopathy Monitoring in Sickle Cell Disease

NHEJ-Mediated Gene Editing, a Versatile Approach to Correct a Variety of Fanconi Anemia Genes in HSCs

Liver-Directed AAV Gene Therapy for Gaucher Disease

The Rare Diseases Pilot for the 100,000 Genomes Project: Findings in Known and New Genes by Analysis of 3,549 Whole Genome Sequenced Samples from Patients and Relatives with Haematological, Haemostasis and Immune Disorders

Persistence of CRISPR/Cas9-Edited Hematopoietic Stem and Progenitor Cells and Reactivation of Fetal Hemoglobin in Nonhuman Primates

Potential Long-Term Treatment of Hemophilia a By Early Postnatal Co-Transplantation of Cord Blood Derived Endothelial Colony-Forming Cells and Placental Mesenchymal Stem Cells

Precision Medicine for Sickle Cell Disease through Whole Genome Sequencing

A Novel Filtering Tool for Detecting Sickle Cell Disease in the Pediatric Population

Gene Therapy for Thalassemia and Sickle Cell Anemia Poses Acceptable Toxicity and Efficacy: Clinical Outcomes in Forty-Seven Cases

Non-Myeloablative Transplant in Severe Sickle Cell Disease Is Safe & Effective. Single Center Experience from Saudi Arabia

Talen Mediated Therapeutic Gene Editing Strategy for ß-Hemoglobinopathies

High Prevalence of Severe Acute Malnutrition in Children with Sickle Cell Anemia over 5 Years of Age in Northern Nigeria

Reprogramming Cell-Signaling By Delivering the Catalytic Domain of Ptpgr Ameliorates Anemia of β-Thalassemia

The NLRP3 Inflammasome in Platelets Is Upregulated in Sickle Cell Disease and Promotes Platelet Aggregation and In Vitro Thrombosis

Novel Lineage Depletion and Manufacturing Allows for Unprecedented Preservation of Autologous Blood Stem Cells for Gene Therapy of Fanconi Anemia Complementation Group Α

TET2 and MSH6: A Novel Role for MMR in the Pathogenesis of Myeloid Malignancies

Altered Blood Gene Transcription in Chuvash Polycythemia and Its Cell Lineage Specificity

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American Society of Hematology

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American Society of Hematology

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