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Journal Articles

Introduction to a review series on gene therapy and gene editing for sickle cell disease and hemophilia

Catherine M. Bollard

Blood (2021) 138 (11): 913.

https://doi.org/10.1182/blood.2021012891

Published: 2021

Journal Articles

Pediatric hemophagocytic lymphohistiocytosis

Scott W. Canna, Rebecca A. Marsh

Blood (2020) 135 (16): 1332–1343.

https://doi.org/10.1182/blood.2019000936

Published: 2020

Journal Articles

Detection and Analysis of Gene Mutations in Patients with Glanzmann's Thrombasthenia

Boyang Sun, Donglei Zhang, Huiyuan Li, Xueqing Dou, Renchi Yang, MD

Blood (2019) 134 (Supplement_1): 2373.

https://doi.org/10.1182/blood-2019-129446

Published: 2019

Journal Articles

The Mystery of "Magic Blood" - Familial Macrothrombocytopenia Associated with a Novel Variant in GP1BA Gene

Zhaohui Liao Arter, MD, Caitlin Yatogo, MS, CGC, Michael C. Chicka, PhD, Jeffrey L. Berenberg, MD

Blood (2019) 134 (Supplement_1): 2380.

https://doi.org/10.1182/blood-2019-122384

Published: 2019

Journal Articles

Incidence of Vaso-Occlusive Crisis Does Not Increase with Achieving Higher Hemoglobin Levels on Voxelotor Treatment or after Discontinuation: Analyses of the HOPE Study

Elliott P. Vichinsky, MD, Paul Telfer, DM, FRCP, Adlette Inati, MD, Margaret Tonda, PharmD, Barbara Tong, PhD, Irene Agodoa, MD, Joshua Lehrer-Graiwer, MD MPhil, FACC, Kenneth I. Ataga, MD

Blood (2019) 134 (Supplement_1): 2313.

https://doi.org/10.1182/blood-2019-129026

Published: 2019

Journal Articles

Modeling of Immune Reconstitution Post CD34 Selected Stem Cell Transplantation in Pediatric Patients with Severe Combined Immune Deficiency

Jean-Sebastien Diana, MD, Naim Bouazza, PhD, Chloé Couzin, MD, Martin Castelle, MD, Alessandra Magnani, MD PhD, Elisa Magrin, PhD, Laure Joseph, MD, Marianne Delville, MD, Francois Lefrere, MD, jean-Marc Treluyer, MD PhD, Capucine Picard, MD PhD, Despina Moshous, MD PhD, Isabelle André, PhD, Stephane Blanche, MD, Benedicte Neven, MD PhD, Marina Cavazzana, MD PhD

Blood (2019) 134 (Supplement_1): 5668.

https://doi.org/10.1182/blood-2019-127974

Published: 2019

Journal Articles

CRISPR/dCas9 (D10A) -Mediated Hb CS Gene Editing and Identification of Genetically Modified Fibroblasts

Lian Yu, Wei Hao Wu, WANG Shunqing, Ken H. Young, Funeng Jiang, PhD

Blood (2019) 134 (Supplement_1): 5754.

https://doi.org/10.1182/blood-2019-129931

Published: 2019

Journal Articles

CD244 Marks Non-Functional Hematopoietic Stem Cells with a Mast Cell Signature after Induction of Endoplasmic Reticulum Stress

Valgardur Sigurdsson, PhD, Shuhei Koide, PhD, Visnja Radulovic, PhD, Els Mansell, PhD, Mark Van Der Garde, MSc, PhD, Tiit Örd, PhD, Tonis Örd, PhD, Kenichi Miharada, PhD

Blood (2019) 134 (Supplement_1): 2474.

https://doi.org/10.1182/blood-2019-128239

Published: 2019

Journal Articles

Predictors of Disease Progression and Survival in Patients with Myelodysplastic Syndrome Secondary to Inherited Bone Marrow Failure Syndromes

Blood (2019) 134 (Supplement_1): 2507.

https://doi.org/10.1182/blood-2019-130129

Published: 2019

Journal Articles

An Autosomal Dominant SCID Form Due to a Gain of Function Mutation in the RAC2 Gene

Chantal Lagresle-Peyrou, PhD, Aurélien Olichon, Hanem Sadek, Philippe Roche, Claudine Tardy, Cindy Da Silva, Alexandrine Garrigue, Alain Fischer, MD PhD, Despina Moshous, MD PhD, Yves Collette, Capucine Picard, MD PhD, Jean-Laurent Casanova, PhD MD, Isabelle André, PhD, Marina Cavazzana, MD PhD

Blood (2019) 134 (Supplement_1): 3742.

https://doi.org/10.1182/blood-2019-126855

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

Phase II Study of Eltrombopag in Subjects with Fanconi Anemia

Daisuke Araki, MD, Ma Evette Barranta, Fariba Chinian, MS, Julie Erb-Alvarez, MPH, Thomas Winkler, MD, Neelam Giri, MD, Andre Larochelle, MD PhD

Blood (2019) 134 (Supplement_1): 3753.

https://doi.org/10.1182/blood-2019-123151

Published: 2019

Journal Articles

Application of High-Throughput Sequencing in the Diagnosis of Inherited Immune-Thrombocytopenia from Children Chronic/Refractory ITP

Ma Jingyao, MD, Zhenping Chen, PhDMD, Huiqing LIU, Jialu Zhang, Hao GU, Runhui Wu, MD PhD

Blood (2019) 134 (Supplement_1): 86.

https://doi.org/10.1182/blood-2019-126771

Published: 2019

Journal Articles

Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor Cells

Agnieszka Czechowicz, MD PhD, Maria Grazia Roncarolo, MD, Brian C Beard, PhD, Ken Law, PhD, Eileen Nicoletti, MD, Paula Río, PhD, Juan A. Bueren, PhD, Jonathan D Schwartz, MD, Sandeep Soni, MBBS

Blood (2019) 134 (Supplement_1): 3350.

https://doi.org/10.1182/blood-2019-127352

Published: 2019

Journal Articles

Economic Burden of End Organ Damage Among Patients with Sickle Cell Disease in the US

Xue Song, Andrew D. Campbell, MD, Ze Cong, PhD, Irene Agodoa, MD, Diane Martinez, DrPH, MPH, Carolyn R. Lew, PhD, Danae Black, Helen Varker, Chris Chan, Sophie M. Lanzkron, MD

Blood (2019) 134 (Supplement_1): 3388.

https://doi.org/10.1182/blood-2019-127173

Published: 2019

Journal Articles

Home-Based Assessment of Patient Reported Outcome Measures Using a Smartphone App Platform: A Feasibility Study

Sherif M. Badawy, MD, Leonardo Barrera, MPH, Graham Zolkowski, BS, Zeinab Alward, MBChB, MPH

Blood (2019) 134 (Supplement_1): 420.

https://doi.org/10.1182/blood-2019-131334

Published: 2019

Journal Articles

Decline in Forced Expiratory Volume in 1 Second in Older Adults with Sickle Cell Disease Is Similar to Adults with Cystic Fibrosis

Brock Hodges, Jodi-Anne T Stewart, BS, Mark Rodeghier, PhD, Karina L Wilkerson, NP, MSN, Adetola A. Kassim, MDMS, Michael R. DeBaun, MDMPH

Blood (2019) 134 (Supplement_1): 1012.

https://doi.org/10.1182/blood-2019-123428

Published: 2019

Journal Articles

Management Strategies and Satisfaction Levels in Patients with Sickle Cell Disease: Interim Results from the International Sickle Cell World Assessment Survey (SWAY)

John James, MSc, Biree Andemariam, MD, Baba PD Inusa, MD, Fuad El-Rassi, Beverly Francis-Gibson, Alecia C. Nero, MD, Caterina P. Minniti, MD, Cassandra Trimnell, BA, Miguel R. Abboud, MD, Jean-Benoît Arlet, Raffaella Colombatti, Mariane de Montalembert, MD PhD, Suman Jain, Wasil Jastaniah, Erfan Nur, MD PhD, Laurie DeBonnett, MD, Ifeyinwa (ify) Osunkwo, MD MPH

Blood (2019) 134 (Supplement_1): 1017.

https://doi.org/10.1182/blood-2019-123643

Published: 2019

Journal Articles

The Effect of Crizanlizumab Plus Standard of Care (SoC) Versus Soc Alone on Renal Function in Patients with Sickle Cell Disease and Chronic Kidney Disease: A Randomized, Multicenter, Open-Label, Phase II Study (STEADFAST)

Kenneth I. Ataga, MD, Santosh L. Saraf, MD, Vimal K. Derebail, MD, Claire C. Sharpe, Adlette Inati, MD, Jeffrey D. Lebensburger, DO, MSPH, Laurie DeBonnett, MD, Shirong Zhang, Pablo Bartolucci, MD PhD

Blood (2019) 134 (Supplement_1): 1018.

https://doi.org/10.1182/blood-2019-124823

Published: 2019

Journal Articles

Blinatumomab in Pediatric Patients with Relapsed/Refractory B-Cell Precursor and Molecularly Resistant Acute Lymphoblastic Leukemia (R/R ALL): Updated Analysis of 110 Patients Treated in an Expanded Access Study (RIALTO)

Franco Locatelli, MD, Gerhard Zugmaier, Peter Bader, MD, Sima Jeha, MD, Paul-Gerhardt Schlegel, Jean-Pierre Bourquin, MD PhD, Rupert Handgretinger, MD, Benoit Brethon, MD, Claudia Rossig, Christiane Chen-Santel

Blood (2019) 134 (Supplement_1): 1294.

https://doi.org/10.1182/blood-2019-121615

Published: 2019

NARROW

Introduction to a review series on gene therapy and gene editing for sickle cell disease and hemophilia

Pediatric hemophagocytic lymphohistiocytosis

Detection and Analysis of Gene Mutations in Patients with Glanzmann's Thrombasthenia

The Mystery of "Magic Blood" - Familial Macrothrombocytopenia Associated with a Novel Variant in GP1BA Gene

Incidence of Vaso-Occlusive Crisis Does Not Increase with Achieving Higher Hemoglobin Levels on Voxelotor Treatment or after Discontinuation: Analyses of the HOPE Study

Modeling of Immune Reconstitution Post CD34 Selected Stem Cell Transplantation in Pediatric Patients with Severe Combined Immune Deficiency

CRISPR/dCas9 (D10A) -Mediated Hb CS Gene Editing and Identification of Genetically Modified Fibroblasts

CD244 Marks Non-Functional Hematopoietic Stem Cells with a Mast Cell Signature after Induction of Endoplasmic Reticulum Stress

Predictors of Disease Progression and Survival in Patients with Myelodysplastic Syndrome Secondary to Inherited Bone Marrow Failure Syndromes

An Autosomal Dominant SCID Form Due to a Gain of Function Mutation in the RAC2 Gene

Characterizing Dyskeratosis Congenita Caused By Parn Mutations in the Zebrafish

Phase II Study of Eltrombopag in Subjects with Fanconi Anemia

Application of High-Throughput Sequencing in the Diagnosis of Inherited Immune-Thrombocytopenia from Children Chronic/Refractory ITP

Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor Cells

Economic Burden of End Organ Damage Among Patients with Sickle Cell Disease in the US

Home-Based Assessment of Patient Reported Outcome Measures Using a Smartphone App Platform: A Feasibility Study

Decline in Forced Expiratory Volume in 1 Second in Older Adults with Sickle Cell Disease Is Similar to Adults with Cystic Fibrosis

Management Strategies and Satisfaction Levels in Patients with Sickle Cell Disease: Interim Results from the International Sickle Cell World Assessment Survey (SWAY)

The Effect of Crizanlizumab Plus Standard of Care (SoC) Versus Soc Alone on Renal Function in Patients with Sickle Cell Disease and Chronic Kidney Disease: A Randomized, Multicenter, Open-Label, Phase II Study (STEADFAST)

Blinatumomab in Pediatric Patients with Relapsed/Refractory B-Cell Precursor and Molecularly Resistant Acute Lymphoblastic Leukemia (R/R ALL): Updated Analysis of 110 Patients Treated in an Expanded Access Study (RIALTO)

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American Society of Hematology

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American Society of Hematology

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