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Journal Articles

Zinc Finger Nuclease-Mediated Disruption of the BCL11A Erythroid Enhancer Results in Enriched Biallelic Editing, Increased Fetal Hemoglobin, and Reduced Sickling in Erythroid Cells Derived from Sickle Cell Disease Patients

Samuel Lessard, PhD, Pauline Rimmele, PhD, Hui Ling, PhD, Kevin Moran, MSc, Benjamin Vieira, BS, Yi-Dong Lin, PhD, Vu Hong, PhD, Andreas Reik, PhD, Denny Dang, Naoya Uchida, MD, John F. Tisdale, MD, Pablo Rendo, MD, Ahmed Daak, PhD, Alexandra Hicks, PhD

Blood (2019) 134 (Supplement_1): 974.

https://doi.org/10.1182/blood-2019-126048

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

Truncated Erythropoietin Receptors Confer an In Vivo Selective Advantage in Gene-Modified Erythroid Cells Expressing Fetal Hemoglobin Due to BCL11A Interference

Naoya Uchida, MD, Claire Drysdale, Morgan Yapundich, Jackson Gamer, Tina Nassehi, Matthew Wielgosz, Francesca Ferrara, PhD, Juan J. Haro-Mora, PhD, Selami Demirci, PhD, Aylin Bonifacino, MS, Allen Krouse, Nathaniel S. Linde, Robert E. Donahue, VMD, Byoung Y. Ryu, PhD, John F. Tisdale, MD

Blood (2019) 134 (Supplement_1): 2063.

https://doi.org/10.1182/blood-2019-122770

Published: 2019

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

Preclinical Evaluation for Engraftment of Gene-Edited CD34+ Cells with a Sickle Cell Disease Mutation in a Rhesus Transplantation Model

Naoya Uchida, MD, Linhong Li, PhD, Tina Nassehi, Morgan Yapundich, Jackson Gamer, Claire Drysdale, Juan J. Haro-Mora, PhD, Selami Demirci, PhD, Aylin Bonifacino, MS, Allen Krouse, Nathaniel S. Linde, Cornell Allen, MS, Suk See De Ravin, MD, PhD, Robert E. Donahue, VMD, Harry L. Malech, MD, Madhusudan Peshwa, PhD, John F. Tisdale, MD

Blood (2019) 134 (Supplement_1): 609.

https://doi.org/10.1182/blood-2019-122727

Published: 2019

Journal Articles

A Single Dose of CD117 Antibody Drug Conjugate Enables Autologous Gene-Modified Hematopoietic Stem Cell Transplant (Gene Therapy) in Nonhuman Primates

Blood (2019) 134 (Supplement_1): 610.

https://doi.org/10.1182/blood-2019-125968

Published: 2019

Journal Articles

Development of a Clinically Applicable Method for High-Efficiency Gene Correction of Plerixafor-Mobilized CD34 + Cells from Patients with Sickle Cell Disease

Naoya Uchida, MD, Linhong Li, Juan J Haro-Mora, PhD, Selami Demirci, PhD, Tina Nassehi, Jackson Gamer, Claire Drysdale, Morgan Yapundich, Cornell Allen, Suk See De Ravin, MD PhD, Harry L Malech, MD, Madhusudan Peshwa, PhD, John F. Tisdale, MD

Blood (2018) 132 (Supplement 1): 2191.

https://doi.org/10.1182/blood-2018-99-111677

Published: 2018

Journal Articles

Intrabone Delivery of CD34+ Cells Using an Optimized Delivery System Does Not Enhance Engraftment in a Rhesus Macaque Model of Hematopoietic Stem Cell Transplantation

Blood (2018) 132 (Supplement 1): 5677.

https://doi.org/10.1182/blood-2018-99-114730

Published: 2018

Journal Articles

Selective Proliferation of Gene-Modified Human Erythroid Cells with Truncated Erythropoietin Receptors

Naoya Uchida, MD PhD, Anna Shvygin, Lydia Raines, Juan Jesus J. Haro Mora, PhD, Selami Demirci, PhD, Matthew M. Hsieh, MD, John F Tisdale, MD

Blood (2017) 130 (Supplement 1): 3338.

https://doi.org/10.1182/blood.V130.Suppl_1.3338.3338

Published: 2017

Journal Articles

Improved ES Cell-Derived Erythroid Cell Generation with Robust β-Globin Production through ES-Sacs

Juan Jesus J. Haro Mora, PhD, Selami Demirci, PhD, John F Tisdale, MD, Naoya Uchida, MD PhD

Blood (2017) 130 (Supplement 1): 3757.

https://doi.org/10.1182/blood.V130.Suppl_1.3757.3757

Published: 2017

Journal Articles

Fetal Hemoglobin Induction with Forced Chromatin Looping in Gene-Modified Erythroid Cells Differentiated from Rhesus Hematopoietic Progenitor Cells

Selami Demirci, PhD, Saurabh Bhardwaj, PhD, Naoya Uchida, MD PhD, Juan J. Haro Mora, PhD, Byoung Y. Ryu, PhD, Gerd A. Blobel, MD PhD, John F Tisdale, MD

Blood (2017) 130 (Supplement 1): 4617.

https://doi.org/10.1182/blood.V130.Suppl_1.4617.4617

Published: 2017

Journal Articles

Bone Marrow Characterization in Sickle Cell Disease: Inflammation and Stress Erythropoiesis Lead to Suboptimal CD34 Recovery Compared to Normal Volunteer Bone Marrow

Alexis Leonard, MD, Aylin Bonifacino, MS, Venina Marcela Dominical, PhD, Anna Conrey, NP, Wynona Coles, Mary Link, RN, Matthew Hsieh, MD, Min Luo, PhD, Naoya Uchida, MD PhD, Francis J. Pierciey, Jr., John F Tisdale, MD

Blood (2017) 130 (Supplement 1): 966.

https://doi.org/10.1182/blood.V130.Suppl_1.966.966

Published: 2017

Journal Articles

Development of a New Generation, Forward-Oriented Therapeutic Vector for Hemoglobin Disorders

Naoya Uchida, MD PhD, Matthew M. Hsieh, MD, Aylin C Bonifacino, Allen E Krouse, Mark E Metzger, Robert E Donahue, John F. Tisdale, MD

Blood (2016) 128 (22): 1172.

https://doi.org/10.1182/blood.V128.22.1172.1172

Published: 2016

Journal Articles

Evaluation of CD4+ Cell Recovery in Vivo Using Single Photon Emission Tomography (SPECT) in Real Time Following CD34+ Cell Transplantation in Rhesus Macaques

Robert E. Donahue, Sharat Srinivasula, Naoya Uchida, MD PhD, Insook Kim, Gorka Duralde, Paula DeGrange, Marisa St. Claire, Richard C. Reba, Aylin C. Bonifacino, Allen E. Krouse, Mark E. Metzger, Chang H. Paik, H. Clifford Lane, John F. Tisdale, MD, Michele Di Mascio

Blood (2014) 124 (21): 1105.

https://doi.org/10.1182/blood.V124.21.1105.1105

Published: 2014

Journal Articles

β-Globin-Expressing Definitive Erythroid Progenitor Cells Generated from Embryonic and Induced Pluripotent Stem Cell-Derived Sacs

Naoya Uchida, MD PhD, Atsushi Fujita, MD PhD, Thomas Winkler, MD, John F. Tisdale, MD

Blood (2014) 124 (21): 2674.

https://doi.org/10.1182/blood.V124.21.2674.2674

Published: 2014

Journal Articles

Discordance Between Peripheral Blood and Tissue CD4+ Lymphocyte Reconstitution In Rhesus Monkeys Following Autologous Hematopoietic CD34+ Cell Transplantation: An In Vivo imaging Study

Robert E. Donahue, Sharat Srinivasula, Naoya Uchida, MD, PhD, Insook Kim, Gorka Duralde, Paula DeGrange, Marisa St. Claire, Richard C. Reba, Aylin C. Bonifacino, Allen E. Krouse, Mark E. Metzger, Chang H. Paik, H. Clifford Lane, John F. Tisdale, MD, Michele Di Mascio

Blood (2013) 122 (21): 2019.

https://doi.org/10.1182/blood.V122.21.2019.2019

Published: 2013

Journal Articles

Generation Of Mature Erythroid Cells From Human Embryonic Stem Cell Derived Sacs and Optimization Of Erythroid Cell Generation

Atsushi Fujita, MD, PhD, Thomas Winkler, MD, John F. Tisdale, MD, Naoya Uchida, MD, PhD

Blood (2013) 122 (21): 2425.

https://doi.org/10.1182/blood.V122.21.2425.2425

Published: 2013

Journal Articles

CD34+CXCR4(CD184)+ Cells Differentiate Into Myeloid Dendritic Cell Progenitors

Robert E. Donahue, Ping Jin, Naoya Uchida, MD, PhD, Aylin C. Bonifacino, Yu Tian, Johanna I. Klinman, Joseph J Mattapallil, Mark E. Metzger, Ann Williams, Daniela Malide, John F. Tisdale, MD, David Stroncek, MD

Blood (2013) 122 (21): 4835.

https://doi.org/10.1182/blood.V122.21.4835.4835

Published: 2013

Journal Articles

Peripheral Blood As a Source Of Cells For Regenerative Medicine Applications In Sickle Cell Disease

Atsushi Fujita, MD, PhD, Courtney D. Fitzhugh, MD, Matthew M. Hsieh, MD, John F. Tisdale, MD, Naoya Uchida, MD, PhD

Blood (2013) 122 (21): 2209.

https://doi.org/10.1182/blood.V122.21.2209.2209

Published: 2013

NARROW

Zinc Finger Nuclease-Mediated Disruption of the BCL11A Erythroid Enhancer Results in Enriched Biallelic Editing, Increased Fetal Hemoglobin, and Reduced Sickling in Erythroid Cells Derived from Sickle Cell Disease Patients

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

Truncated Erythropoietin Receptors Confer an In Vivo Selective Advantage in Gene-Modified Erythroid Cells Expressing Fetal Hemoglobin Due to BCL11A Interference

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

Preclinical Evaluation for Engraftment of Gene-Edited CD34+ Cells with a Sickle Cell Disease Mutation in a Rhesus Transplantation Model

A Single Dose of CD117 Antibody Drug Conjugate Enables Autologous Gene-Modified Hematopoietic Stem Cell Transplant (Gene Therapy) in Nonhuman Primates

Development of a Clinically Applicable Method for High-Efficiency Gene Correction of Plerixafor-Mobilized CD34 + Cells from Patients with Sickle Cell Disease

Intrabone Delivery of CD34+ Cells Using an Optimized Delivery System Does Not Enhance Engraftment in a Rhesus Macaque Model of Hematopoietic Stem Cell Transplantation

Selective Proliferation of Gene-Modified Human Erythroid Cells with Truncated Erythropoietin Receptors

Improved ES Cell-Derived Erythroid Cell Generation with Robust β-Globin Production through ES-Sacs

Fetal Hemoglobin Induction with Forced Chromatin Looping in Gene-Modified Erythroid Cells Differentiated from Rhesus Hematopoietic Progenitor Cells

Bone Marrow Characterization in Sickle Cell Disease: Inflammation and Stress Erythropoiesis Lead to Suboptimal CD34 Recovery Compared to Normal Volunteer Bone Marrow

Development of a New Generation, Forward-Oriented Therapeutic Vector for Hemoglobin Disorders

Evaluation of CD4+ Cell Recovery in Vivo Using Single Photon Emission Tomography (SPECT) in Real Time Following CD34+ Cell Transplantation in Rhesus Macaques

β-Globin-Expressing Definitive Erythroid Progenitor Cells Generated from Embryonic and Induced Pluripotent Stem Cell-Derived Sacs

Discordance Between Peripheral Blood and Tissue CD4+ Lymphocyte Reconstitution In Rhesus Monkeys Following Autologous Hematopoietic CD34+ Cell Transplantation: An In Vivo imaging Study

Generation Of Mature Erythroid Cells From Human Embryonic Stem Cell Derived Sacs and Optimization Of Erythroid Cell Generation

CD34+CXCR4(CD184)+ Cells Differentiate Into Myeloid Dendritic Cell Progenitors

Peripheral Blood As a Source Of Cells For Regenerative Medicine Applications In Sickle Cell Disease

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American Society of Hematology

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ASH Publications

American Society of Hematology

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