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Journal Articles

Adenosine Base Editing of γ-Globin Promoters Induces Fetal Hemoglobin and Inhibit Erythroid Sickling

Thiyagaraj Mayuranathan, Jonathan S. Yen, PhD, Gregory A. Newby, PhD, Yu Yao, MD, Shaina N. Porter, PhD, Kaitly J. Woodard, BS, Jingjing Zhang, MS, Kalin Mayberry, BS, Akshay Sharma, MBBS, Shondra M. preutt-Miller, PhD, John F. Tisdale, MD, Shengdar Q. Tsai, PhD, David R. Liu, PhD, Mitchell J. Weiss, MD PhD

Blood (2020) 136 (Supplement 1): 21–22.

https://doi.org/10.1182/blood-2020-141498

Published: 2020

Journal Articles

Base Editing Eliminates the Sickle Cell Mutation and Pathology in Hematopoietic Stem Cells Derived Erythroid Cells

Jonathan S Yen, PhD, Gregory A. Newby, PhD, Thiyagaraj Mayuranathan, Shaina N. Porter, PhD, Yu Yao, MD, Kaitly J. Woodard, BS, Kalin Mayberry, BS, Kelcee Everette, Jingjing Zhang, MS, Jordana M Henderson, MS, Anton P Mccaffrey, Jonathan Yen, John F. Tisdale, MD, Shengdar Q. Tsai, PhD, David R. Liu, PhD, Mitchell J. Weiss, MD PhD

Blood (2020) 136 (Supplement 1): 13–14.

https://doi.org/10.1182/blood-2020-139016

Published: 2020

Journal Articles

Control of Fetal Hemoglobin Levels By NFI Transcription Factors

Kunhua Qin, Peng Huang, PhD, Anran Huang, BS, Ruopeng Feng, PhD, Thiyagaraj Mayuranathan, Cheryl A. Keller, PhD, Belinda Giardine, Ross C. Hardison, PhD, Mitchell J. Weiss, MD PhD, Junwei Shi, PhD, Gerd A. Blobel, MDPhD

Blood (2020) 136 (Supplement 1): 54.

https://doi.org/10.1182/blood-2020-136167

Published: 2020

Journal Articles

ZNF410 Uniquely Activates the NuRD Component CHD4 to Silence Fetal Hemoglobin Expression

Blood (2020) 136 (Supplement 1): 54.

https://doi.org/10.1182/blood-2020-137564

Published: 2020

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

Safe and Efficient Peripheral Blood Stem Cell Collection in Patients with Sickle Cell Disease Using Plerixafor

Alexis Leonard, MD, Naoya Uchida, MD, David F. Stroncek, MD, Sandhya R. Panch, MDMPH, Kamille West, Eoghan George Molloy, MD, Tom Hughes, Pharm.D., Sara Hauffe, RN, Tiffani Taylor, Courtney D. Fitzhugh, MD, Jane S. Hankins, MD MS, Megan Wilson, MSW, CCRP, Akshay Sharma, MBBS, Shengdar Q. Tsai, PhD, Mitchell J. Weiss, MD, PhD, Matthew Hsieh, MD, John F. Tisdale, MD

Blood (2019) 134 (Supplement_1): 1964.

https://doi.org/10.1182/blood-2019-126091

Published: 2019

Journal Articles

Lentiviral Gene Therapy with Low Dose Busulfan for Infants with X-SCID Results in the Development of a Functional Normal Immune System: Interim Results of an Ongoing Phase I/II Clinical Study

Blood (2019) 134 (Supplement_1): 2058.

https://doi.org/10.1182/blood-2019-126746

Published: 2019

Journal Articles

Disease Modeling and Phenotype Rescue Using Inducible Pluripotent Stem Cells from Patients with Diamond-Blackfan Anemia

Senthil Velan Bhoopalan, MD PhD, Min-Joon Han, PhD, Steven Ellis, PhD, Harry Lesmana, MD, Jeremie H. Estepp, MD, Yoonjeong Jang, DVM,PhD, Mitchell J. Weiss, MD PhD, Marcin W. Wlodarski, MD PhD

Blood (2019) 134 (Supplement_1): 2496.

https://doi.org/10.1182/blood-2019-131354

Published: 2019

Journal Articles

Regulation of Fetal Hemoglobin through the Insulin Signaling Pathway

Alireza Paikari, MD, Yankai Zhang, PhD, Alicia Chang, MD, Ankush Goyal, MA, Evadnie Rampersaud, PhD, Jonathan M. Flanagan, PhD, Mitchell J. Weiss, MD, PhD, Vivien A. Sheehan, MD PhD

Blood (2019) 134 (Supplement_1): 811.

https://doi.org/10.1182/blood-2019-131313

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

The DNA Methylation Maintenance Protein UHRF1 Regulates Fetal Globin Expression Independent of HBG Promoter DNA Methylation

Ruopeng Feng, PhD, Phillip A Doerfler, PhD, Yu Yao, MD, Xing Tang, PhD, Yong-Dong Wang, PhD, Chunliang Li, PhD, David E. Root, PhD, Yong Cheng, PhD, Mitchell J. Weiss, MD PhD

Blood (2018) 132 (Supplement 1): 410.

https://doi.org/10.1182/blood-2018-99-115589

Published: 2018

Journal Articles

Activation of ULK1 Kinase Mediates Clearance of Free Alpha-Globin in Human Beta-Thalassemic Erythroblasts

Christophe Lechauve, PhD, Julia Keith, Eugene Khandros, MD PhD, Stephanie Fowler, Kalin Mayberry, Abdullah Freiwan, Christopher Thom, MD PhD, Jingjing Zhang, Heather Tillman, DVM, PhD, Mondira Kundu, MD PhD, Irene Motta, MD, Maria Domenica Cappellini, MD, Mitchell J. Weiss, MD PhD

Blood (2018) 132 (Supplement 1): 411.

https://doi.org/10.1182/blood-2018-99-113901

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

Modelling the Progression of a Preleukemic Stage to Overt Leukemia in Children with Down Syndrome

Blood (2018) 132 (Supplement 1): 543.

https://doi.org/10.1182/blood-2018-99-116661

Published: 2018

Journal Articles

FBXO11 Activates Erythroid Gene Transcription By Degrading Heterochromatin-Associated Protein BAHD1

Peng Xu, PhD, Daniel C. Scott, PhD, Xing Tang, PhD, Yu Yao, MD, Yong-Dong Wang, PhD, Wenjian Bi, PhD, Lance E. Palmer, PhD, Ruopeng Feng, PhD, Arno F. Alpi, PhD, Chunliang Li, PhD, Yong Cheng, PhD, Brenda A. Schulman, PhD, Mitchell J. Weiss, MD PhD

Blood (2018) 132 (Supplement 1): 529.

https://doi.org/10.1182/blood-2018-99-117362

Published: 2018

Journal Articles

Accurate Prediction of RH Genotypes Using Whole Genome Sequencing Data

Yan Zheng, MD PhD, Ti-Cheng Chang, PhD, Gang Wu, PhD, Jane S. Hankins, MD MS, Mitchell J. Weiss, MD PhD, Connie M. Westhoff, PhD, Stella T. Chou, MD

Blood (2018) 132 (Supplement 1): 2332.

https://doi.org/10.1182/blood-2018-99-119681

Published: 2018

Journal Articles

Insulin-like Growth Factor Binding Protein-3 (IGFBP3) Induces Fetal Hemoglobin in Hematopoietic Stem and Progenitor Cells from Patients with Sickle Cell Anemia

Alireza Paikari, MD, Tian Mi, PhD, Yankai Zhang, PhD, Evadnie Rampersaud, PhD, Guolian Kang, PhD, Gang Wu, PhD, Jane S. Hankins, MD MS, Jeremie H. Estepp, MD, Pavel Sumazin, PhD, Mitchell J. Weiss, MD PhD, Vivien A Sheehan, MD PhD

Blood (2018) 132 (Supplement 1): 722.

https://doi.org/10.1182/blood-2018-99-115383

Published: 2018

Journal Articles

Data Access and Interactive Visualization of Whole Genome Sequence of Sickle Cell Patients within the St. Jude Cloud

Blood (2018) 132 (Supplement 1): 723.

https://doi.org/10.1182/blood-2018-99-116597

Published: 2018

Journal Articles

Ulk1 Kinase Mediates Clearance of Free α-Globin in β-Thalassemia

Christophe Lechauve, PhD, Eugene Khandros, MD PhD, Christopher Thom, MD PhD, Abdullah Freiwan, Stephanie Fowler, Kalin Mayberry, Julia Keith, Heather Tillman, DVM, PhD, Mondira Kundu, MD PhD, Mitchell J. Weiss, MD PhD

Blood (2017) 130 (Supplement 1): 285.

https://doi.org/10.1182/blood.V130.Suppl_1.285.285

Published: 2017

Journal Articles

Interim Results from a Phase I/II Clinical Gene Therapy Study for Newly Diagnosed Infants with X-Linked Severe Combined Immunodeficiency Using a Safety-Modified Lentiviral Vector and Targeted Reduced Exposure to Busulfan

Blood (2017) 130 (Supplement 1): 523.

https://doi.org/10.1182/blood.V130.Suppl_1.523.523

Published: 2017

NARROW

Adenosine Base Editing of γ-Globin Promoters Induces Fetal Hemoglobin and Inhibit Erythroid Sickling

Base Editing Eliminates the Sickle Cell Mutation and Pathology in Hematopoietic Stem Cells Derived Erythroid Cells

Control of Fetal Hemoglobin Levels By NFI Transcription Factors

ZNF410 Uniquely Activates the NuRD Component CHD4 to Silence Fetal Hemoglobin Expression

CRISPR-Cas9 Genome Editing of γ-Globin Promoters in Human Hematopoietic Stem Cells to Induce Erythrocyte Fetal Hemoglobin for Treatment of β-Hemoglobinopathies

Safe and Efficient Peripheral Blood Stem Cell Collection in Patients with Sickle Cell Disease Using Plerixafor

Lentiviral Gene Therapy with Low Dose Busulfan for Infants with X-SCID Results in the Development of a Functional Normal Immune System: Interim Results of an Ongoing Phase I/II Clinical Study

Disease Modeling and Phenotype Rescue Using Inducible Pluripotent Stem Cells from Patients with Diamond-Blackfan Anemia

Regulation of Fetal Hemoglobin through the Insulin Signaling Pathway

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

The DNA Methylation Maintenance Protein UHRF1 Regulates Fetal Globin Expression Independent of HBG Promoter DNA Methylation

Activation of ULK1 Kinase Mediates Clearance of Free Alpha-Globin in Human Beta-Thalassemic Erythroblasts

Precision Medicine for Sickle Cell Disease through Whole Genome Sequencing

Modelling the Progression of a Preleukemic Stage to Overt Leukemia in Children with Down Syndrome

FBXO11 Activates Erythroid Gene Transcription By Degrading Heterochromatin-Associated Protein BAHD1

Accurate Prediction of RH Genotypes Using Whole Genome Sequencing Data

Insulin-like Growth Factor Binding Protein-3 (IGFBP3) Induces Fetal Hemoglobin in Hematopoietic Stem and Progenitor Cells from Patients with Sickle Cell Anemia

Data Access and Interactive Visualization of Whole Genome Sequence of Sickle Cell Patients within the St. Jude Cloud

Ulk1 Kinase Mediates Clearance of Free α-Globin in β-Thalassemia

Interim Results from a Phase I/II Clinical Gene Therapy Study for Newly Diagnosed Infants with X-Linked Severe Combined Immunodeficiency Using a Safety-Modified Lentiviral Vector and Targeted Reduced Exposure to Busulfan

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American Society of Hematology

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ASH Publications

American Society of Hematology

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