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Journal Articles

Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia and Sickle Cell Disease: Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-CAS9-Modified CD34+ Hematopoietic Stem and Progenitor Cells

Blood (2020) 136 (Supplement 1): 3–4.

https://doi.org/10.1182/blood-2020-139575

Published: 2020

Journal Articles

Targeting Transcriptional Co-Activator PGC-1a for the Treatment of Sickle Cell Disease

Alawi Habara, MBBS, MSc, Cuong LE, PhD, David H.K. Chui, MD, Martin H. Steinberg, MD, Shuaiying Cui, PhD

Blood (2019) 134 (Supplement_1): 2254.

https://doi.org/10.1182/blood-2019-123959

Published: 2019

Journal Articles

Regulating Transcriptional Co-Activator PGC-1α for the Treatment of Sickle Cell Disease

Alawi Habara, MBBS, MSc, Cuong LE, PhD, George J Murphy, PhD, David H.K. Chui, MD, Martin H. Steinberg, MD, Shuaiying Cui, PhD

Blood (2018) 132 (Supplement 1): 2358.

https://doi.org/10.1182/blood-2018-99-111022

Published: 2018

Journal Articles

LSD1 Inhibitors Induce Fetal Hemoglobin in Primary Human Erythroid Cells

Cuong LE, PhD, Greggory Myers, Alawi Habara, MBBS, MSc, David H.K. Chui, MD, Martin H. Steinberg, MD, James Douglas Engel, PhD, Shuaiying Cui, PhD

Blood (2018) 132 (Supplement 1): 1066.

https://doi.org/10.1182/blood-2018-99-117675

Published: 2018

Journal Articles

Biomarker Signatures of Sickle Cell Disease Severity

Mengtian Du, BA, Sarah Van Ness, BA, Victor R. Gordeuk, MD, Mark T Gladwin, MD, Paola Sebastiani, PhD, Martin H. Steinberg, MD

Blood (2017) 130 (Supplement 1): 690.

https://doi.org/10.1182/blood.V130.Suppl_1.690.690

Published: 2017

Journal Articles

Induced Pluripotent Stem Cell (iPSC)-Based Mapping of Globin Expression throughout Human Erythropoietic Development

Kim Vanuytsel, PhD, Taylor Matte, Amy Leung, PhD, Zaw Htut Naing, Tasha A. Morrison, MPH,BA, David H.K. Chui, MD, Martin H. Steinberg, MD, George J Murphy, PhD

Blood (2017) 130 (Supplement 1): 946.

https://doi.org/10.1182/blood.V130.Suppl_1.946.946

Published: 2017

Journal Articles

SIRT1 Increases the Expression of Fetal Hemoglobin Genes in Human Adult and Cord Blood Erythroblasts

Yan Dai, PhD, Tyngwei Chen, Heba Ijaz, Elizabeth Cho, Ryo Kurita, PhD, Yukio Nakamura, MD PhD, Martin H. Steinberg, MD

Blood (2017) 130 (Supplement 1): 287.

https://doi.org/10.1182/blood.V130.Suppl_1.287.287

Published: 2017

Journal Articles

A Long Noncoding RNA from the HBS1L-MYB Intergenic Region on Chr6q23 Regulates Human Fetal Hemoglobin Expression

Tasha A. Morrison, MPH,BA, Ibifiri Wilcox, BS, Hong-Yuan Luo, PhD, John Farrell, PhD, Ryo Kurita, PhD, Yukio Nakamura, MD PhD, George J Murphy, PhD, Shuaiying Cui, PhD, Martin H. Steinberg, MD, David H.K. Chui, MD

Blood (2017) 130 (Supplement 1): 288.

https://doi.org/10.1182/blood.V130.Suppl_1.288.288

Published: 2017

Journal Articles

In Vivo Effects of LSD1 Inhibition By Small Chemical Inhibitors in Sickle Cell Mice

Cuong Le, PhD, Greggory Myers, Alawi Habara, Natee Jearawiriyapaisarn, PhD, Mary Lee, PhD, David H.K. Chui, MD, Martin H. Steinberg, MD, James Engel, PhD, Shuaiying Cui, PhD

Blood (2017) 130 (Supplement 1): 968.

https://doi.org/10.1182/blood.V130.Suppl_1.968.968

Published: 2017

Journal Articles

A Candidate Trans-Acting Modulator of Fetal Hemoglobin Gene Expression in the Arab-Indian Haplotype of Sickle Cell Anemia

Blood (2015) 126 (23): 409.

https://doi.org/10.1182/blood.V126.23.409.409

Published: 2015

Journal Articles

Association of FOXO3A Polymorphisms with Hematocrit, LDH and Longevity in Patients with Sickle Cell Anemia from CSSCD, Walk-Phasst, and PUSH Clinical Trials

Harold T Bae, PhD MS, Paola Sebastiani, PhD, Victor R. Gordeuk, MD, Yingze Zhang, PhD, Martin H. Steinberg, MD, Gregory J Kato, MD

Blood (2015) 126 (23): 2176.

https://doi.org/10.1182/blood.V126.23.2176.2176

Published: 2015

Journal Articles

Polymorphisms Associated with the Arab-Indian Haplotype of Sickle Cell Anemia Are Candidate Fetal Hemoglobin Gene Modulators

Vinod Vathipadiekal, PhD, Abdulrahman Alsultan, MD, John Farrell, PhD, A.M Al-Rubaish, Fahad Al-Muhanna, Z Naserullah, A Alsuliman, P.K. Patra, Jacqueline Milton, Lindsay Farrer, David H.K. Chui, MD, Amein Al-Ali, Paola Sebastiani, PhD, Martin H. Steinberg, MD

Blood (2015) 126 (23): 3388.

https://doi.org/10.1182/blood.V126.23.3388.3388

Published: 2015

Journal Articles

Characterization of a Transcript Found within the HBS1L-MYB Intergenic Region

Tasha Morrison, MPH,BA, Ibi Wilcox, BA, Hong Yuan Luo, MB, PhD, Martin H. Steinberg, MD, David H.K. Chui, MD

Blood (2014) 124 (21): 2684.

https://doi.org/10.1182/blood.V124.21.2684.2684

Published: 2014

Journal Articles

Assessment of HbF QTLs Affecting Disease Severity and Genetic Analysis in Patients Homozygous for Codon 8 (–AA) β 0 -Thalassemia Mutation

Zhihua Jiang, PhD, Shengwen Huang, MD PhD, Hong Yuan Luo, MB, PhD, Nejat Akar, MD, A.Nazli Basak, MD, Nasir Al-Allawi, MD PhD, Sule Unal, MD, Fatma Gumruk, MDProf, Lance Davis, MD, Tasha Morrison, MPH,BA, Andrew Campbell, MD, Patrick G. Gallagher, MD, Bernard G Forget, MD, Martin H. Steinberg, MD, David H.K. Chui, MD

Blood (2014) 124 (21): 2690.

https://doi.org/10.1182/blood.V124.21.2690.2690

Published: 2014

Journal Articles

Genes Associated with Alloimmunization to Blood Group Antigens in Sickle Cell Disease

Jacqueline N. Milton, PhD, Allison E. Ashley-Koch, PhD, Melanie E. Garrett, MS, Karen L. Soldano, B.S., Eugene P. Orringer, MD, Paola Sebastiani, PhD, Daniel A. Dworkis, MD PhD, Karen Quillen, MD MPH, Martin H. Steinberg, MD, Marilyn J. Telen, MD

Blood (2014) 124 (21): 762.

https://doi.org/10.1182/blood.V124.21.762.762

Published: 2014

Journal Articles

Analysis of (δβ) 0 Thalassemia and HPFH Deletions Suggest a Hierarchy of Cis-Acting Elements Regulating Fetal Hemoglobin Gene Expression.

Heather L Edward, BA, Tasha Morrison, MPH,BA, Jacqueline N Milton, MS, Hong-yuan Luo, MB, PhD, Lance Davis, MD, Bernard G Forget, MD, Martin H. Steinberg, MD, David H.K. Chui, MD

Blood (2014) 124 (21): 54.

https://doi.org/10.1182/blood.V124.21.54.54

Published: 2014

Journal Articles

BCL11A enhancer Haplotypes Are Associated with the Distribution of HbF in Arab-Indian and African Haplotype Sickle Cell Anemia but Not the Different Population Levels of HbF

Paola Sebastiani, PhD, John J. Farrell, PhD, Shuai Wang, MS, Heather L Edward, BA, Heather M. Shappell, BS, Harold T. Bae, PhD, Clinton T. Baldwin, PhD, A. M. Al-Rubaish, MD, Zaki Naserullah, MD, Ahmed Alsuliman, MD, Pradeep K. Patra, PhD, Lindsay A. Farrer, PhD, David H.K. Chui, MD, Abdulrahman Alsultan, MD, Duyen A. Ngo, MD, Haifa Kathrin Al-Ali, MD, Martin H. Steinberg, MD

Blood (2014) 124 (21): 4066.

https://doi.org/10.1182/blood.V124.21.4066.4066

Published: 2014

Journal Articles

Multisystem, Induced Pluripotent Stem Cell (iPSC) Modeling Reveals a Role for Growth Differentiation Factors (GDFs) in the Etiology of β Thalassemia and Ineffective Erythropoiesis

Liz B. Wang, Andreia Gianotti Sommer, PhD, Amy Leung, PhD, Seonmi Park, David H.K. Chui, MD, Martin H. Steinberg, MD, Gustavo Mostoslavsky, MD, PhD, George J. Murphy, PhD

Blood (2014) 124 (21): 4039.

https://doi.org/10.1182/blood.V124.21.4039.4039

Published: 2014

Journal Articles

Neonatal Hemolytic Anemia and (δβ) 0 -Thalassemia Caused By Novel Deletions Involving The β-Globin Gene Cluster

Morrison Tasha, MPH, Hong-yuan Luo, MB, PhD, Lance Davis, MD, Roger Theberge, PhD, Mark E McComb, PhD, Catherine E. Costello, PhD, Jeanne Boudreaux, MD, Zeqiu Han, MD, Martin H. Steinberg, MD, Bernard G. Forget, MD, David H.K. Chui, MD

Blood (2013) 122 (21): 3452.

https://doi.org/10.1182/blood.V122.21.3452.3452

Published: 2013

Journal Articles

Patients Homozygous For Codon 8 (–AA) Frame-Shift β 0 -Thalassemia Mutation With Markedly Increased HbF

Zhihua Jiang, PhD, Hong-yuan Luo, MB, PhD, Nasir Al-Allawi, MD, PhD, Sule Unal, MD, Fatma Gumruk, MD, Leonor Osorio-Almeida, PhD, Lance Davis, MD, Tasha Morrison, MPH, Andrew D. Campbell, MD, Martin H. Steinberg, MD, Patrick G Gallagher, MD, Bernard G. Forget, MD, David H.K. Chui, MD

Blood (2013) 122 (21): 3455.

https://doi.org/10.1182/blood.V122.21.3455.3455

Published: 2013

NARROW

Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia and Sickle Cell Disease: Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-CAS9-Modified CD34+ Hematopoietic Stem and Progenitor Cells

Targeting Transcriptional Co-Activator PGC-1a for the Treatment of Sickle Cell Disease

Regulating Transcriptional Co-Activator PGC-1α for the Treatment of Sickle Cell Disease

LSD1 Inhibitors Induce Fetal Hemoglobin in Primary Human Erythroid Cells

Biomarker Signatures of Sickle Cell Disease Severity

Induced Pluripotent Stem Cell (iPSC)-Based Mapping of Globin Expression throughout Human Erythropoietic Development

SIRT1 Increases the Expression of Fetal Hemoglobin Genes in Human Adult and Cord Blood Erythroblasts

A Long Noncoding RNA from the HBS1L-MYB Intergenic Region on Chr6q23 Regulates Human Fetal Hemoglobin Expression

In Vivo Effects of LSD1 Inhibition By Small Chemical Inhibitors in Sickle Cell Mice

A Candidate Trans-Acting Modulator of Fetal Hemoglobin Gene Expression in the Arab-Indian Haplotype of Sickle Cell Anemia

Association of FOXO3A Polymorphisms with Hematocrit, LDH and Longevity in Patients with Sickle Cell Anemia from CSSCD, Walk-Phasst, and PUSH Clinical Trials

Polymorphisms Associated with the Arab-Indian Haplotype of Sickle Cell Anemia Are Candidate Fetal Hemoglobin Gene Modulators

Characterization of a Transcript Found within the HBS1L-MYB Intergenic Region

Assessment of HbF QTLs Affecting Disease Severity and Genetic Analysis in Patients Homozygous for Codon 8 (–AA) β 0 -Thalassemia Mutation

Genes Associated with Alloimmunization to Blood Group Antigens in Sickle Cell Disease

Analysis of (δβ) 0 Thalassemia and HPFH Deletions Suggest a Hierarchy of Cis-Acting Elements Regulating Fetal Hemoglobin Gene Expression.

BCL11A enhancer Haplotypes Are Associated with the Distribution of HbF in Arab-Indian and African Haplotype Sickle Cell Anemia but Not the Different Population Levels of HbF

Multisystem, Induced Pluripotent Stem Cell (iPSC) Modeling Reveals a Role for Growth Differentiation Factors (GDFs) in the Etiology of β Thalassemia and Ineffective Erythropoiesis

Neonatal Hemolytic Anemia and (δβ) 0 -Thalassemia Caused By Novel Deletions Involving The β-Globin Gene Cluster

Patients Homozygous For Codon 8 (–AA) Frame-Shift β 0 -Thalassemia Mutation With Markedly Increased HbF

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American Society of Hematology

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ASH Publications

American Society of Hematology

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