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Journal Articles

CD117 Antibody Conditioning and Multiplex Base Editing Enable Rapid and Robust Fetal Hemoglobin Reactivation in a Rhesus Autologous Transplantation Model

Blood (2024) 144 (Supplement 1): 516.

https://doi.org/10.1182/blood-2024-204403

Published: 2024

Journal Articles

CD117 Antibody-Drug Conjugate Conditioning Allows Efficient Engraftment of Gene-Modified CD34+ Cells with Fertility Preservation in a Rhesus Gene Therapy Model

Blood (2023) 142 (Supplement 1): 486.

https://doi.org/10.1182/blood-2023-177815

Published: 2023

Journal Articles

Optimization of Lentiviral ß-Globin Vectors in a Forward Orientation to Enhance Therapeutic Effectiveness for Sickle Cell Disease

Jennifer Okalova, Jordan Shields Alexander, Christopher Chambliss, Zhongyu Zhu, Yanping Xie, Matthew Addington-Hall, Oxana Slessareva, Tony Luo, Beatrix Ferencz, Ibeawuchi Oparaocha, Naoya Uchida, John F. Tisdale, Boro Dropulic, H. Trent Spencer, Rimas J Orentas, David R Archer

Blood (2023) 142 (Supplement 1): 5002.

https://doi.org/10.1182/blood-2023-187506

Published: 2023

Journal Articles

A macaque clonal hematopoiesis model demonstrates expansion of TET2-disrupted clones and utility for testing interventions

Blood (2022) 140 (16): 1774–1789.

https://doi.org/10.1182/blood.2021014875

Published: 2022

Includes: Supplemental data

Journal Articles

CD117 Antibody Drug Conjugate-Based Conditioning Allows for Efficient Engraftment of Gene-Modified CD34+ Cells in a Rhesus Gene Therapy Model

Naoya Uchida, Ulana Stasula, Malikiya Hinds, Paula Germino-Watnick, Allen E. Krouse, N Seth Linde, Aylin C. Bonifacino, Kellie Latimer, Prashant Bhattarai, Nicholas C. Yoder, Rahul Palchaudhuri, Qing Li, Kirk Bertelsen, Lisa Olson, Robert E. Donahue, John F. Tisdale

Blood (2021) 138 (Supplement 1): 560.

https://doi.org/10.1182/blood-2021-145524

Published: 2021

Journal Articles

Optimization of Autologous Hematopoietic Progenitor Stem Cell Apheresis Collection from Plerixafor-Mobilized Patients with Sickle Cell Disease

Blood (2021) 138 (Supplement 1): 1770.

https://doi.org/10.1182/blood-2021-146669

Published: 2021

Journal Articles

Combined +58 and +55 BCL11A enhancer Editing Yields Exceptional Efficiency, Specificity and HbF Induction in Human and NHP Preclinical Models

Blood (2021) 138 (Supplement 1): 1852.

https://doi.org/10.1182/blood-2021-152634

Published: 2021

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

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

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

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

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

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

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

NARROW

CD117 Antibody Conditioning and Multiplex Base Editing Enable Rapid and Robust Fetal Hemoglobin Reactivation in a Rhesus Autologous Transplantation Model

CD117 Antibody-Drug Conjugate Conditioning Allows Efficient Engraftment of Gene-Modified CD34+ Cells with Fertility Preservation in a Rhesus Gene Therapy Model

Optimization of Lentiviral ß-Globin Vectors in a Forward Orientation to Enhance Therapeutic Effectiveness for Sickle Cell Disease

A macaque clonal hematopoiesis model demonstrates expansion of TET2-disrupted clones and utility for testing interventions

CD117 Antibody Drug Conjugate-Based Conditioning Allows for Efficient Engraftment of Gene-Modified CD34+ Cells in a Rhesus Gene Therapy Model

Optimization of Autologous Hematopoietic Progenitor Stem Cell Apheresis Collection from Plerixafor-Mobilized Patients with Sickle Cell Disease

Combined +58 and +55 BCL11A enhancer Editing Yields Exceptional Efficiency, Specificity and HbF Induction in Human and NHP Preclinical Models

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

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

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

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

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

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

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

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American Society of Hematology

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ASH Publications

American Society of Hematology

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