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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

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

The Impact of CD45-Antibody-Drug Conjugate Conditioning on Clonal Dynamics and Immune Tolerance Post HSPC Transplantation in Rhesus Macaques

Taha Bartu Hayal, Chuanfeng Wu, Diana Abraham, Selami Demirci, Rahul Palchaudhuri, Leanne Lanieri, Anjali Bhat, Tiffany Tate, Zhuyan Guo, So Gun Hong, Lisa Olson, Robert Donahue, John F. Tisdale, Cynthia E. Dunbar

Blood (2023) 142 (Supplement 1): 3419.

https://doi.org/10.1182/blood-2023-182066

Published: 2023

Journal Articles

Efficacy, Safety, and Health-Related Quality of Life (HRQOL) in Patients with Sickle Cell Disease (SCD) Who Have Received Lovotibeglogene Autotemcel (Lovo-cel) Gene Therapy: Up to 60 Months of Follow-up

Julie Kanter, Alexis A. Thompson, Janet L. Kwiatkowski, Suhag Parikh, Markus Mapara, Stacey Rifkin-Zenenberg, Banu Aygun, Kimberly A. Kasow, Ashish O. Gupta, Lixin Zhang, Emily Sheldon-Waniga, Meghan Gallagher, Katiana Gruppioni, Anjulika Chawla, Heidi Elliot, Francis J. Pierciey, Mark C. Walters, John F. Tisdale

Blood (2023) 142 (Supplement 1): 1051.

https://doi.org/10.1182/blood-2023-174229

Published: 2023

Journal Articles

Lovo-cel (bb1111) Gene Therapy for Sickle Cell Disease: Updated Clinical Results and Investigations into Two Cases of Anemia from Group C of the Phase 1/2 HGB-206 Study

Mark C. Walters, Alexis A. Thompson, Janet L. Kwiatkowski, Suhag Parikh, Markus Y. Mapara, Stacey Rifkin-Zenenberg, Banu Aygun, Kimberly A. Kasow, Alex Miller, Lixin Zhang, Anjulika Chawla, Elizabeth R. Macari, Francis J. Pierciey, Jr., John F. Tisdale, Julie Kanter

Blood (2022) 140 (Supplement 1): 26–28.

https://doi.org/10.1182/blood-2022-162288

Published: 2022

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

Polyclonality Strongly Correlates with Biological Outcomes and Is Significantly Increased Following Improvements to the Phase 1/2 HGB-206 Protocol and Manufacturing of LentiGlobin for Sickle Cell Disease (SCD; bb1111) Gene Therapy (GT)

Blood (2021) 138 (Supplement 1): 561.

https://doi.org/10.1182/blood-2021-147760

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

Sustained Improvements in Patient-Reported Quality of Life up to 24 Months Post-Treatment with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Mark C. Walters, John F. Tisdale, Markus Y. Mapara, Lakshmanan Krishnamurti, Janet L. Kwiatkowski, Banu Aygun, Kimberly A. Kasow, Stacey Rifkin-Zenenberg, Jennifer Jaroscak, Diana Garbinsky, Costel Chirila, Meghan E. Gallagher, Xinyan Zhang, Pei-Ran Ho, Alexis A. Thompson, Julie Kanter

Blood (2021) 138 (Supplement 1): 7.

https://doi.org/10.1182/blood-2021-146905

Published: 2021

Journal Articles

Gene therapy for sickle cell disease: moving from the bench to the bedside

Allistair A. Abraham, John F. Tisdale

Blood (2021) 138 (11): 932–941.

https://doi.org/10.1182/blood.2019003776

Published: 2021

Journal Articles

Dietary iron restriction improves markers of disease severity in murine sickle cell anemia

Nermi L. Parrow, Pierre-Christian Violet, Nisha Ajit George, Faris Ali, Shivam Bhanvadia, Ryan Wong, John F. Tisdale, Courtney Fitzhugh, Mark Levine, Swee Lay Thein, Robert E. Fleming

Blood (2021) 137 (11): 1553–1555.

https://doi.org/10.1182/blood.2020006919

Published: 2021

Includes: Supplemental data

Journal Articles

Improvements in Health-Related Quality of Life for Patients Treated with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Julie Kanter, MD, John F. Tisdale, MD, Markus Y Mapara, MD PhD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Ren Chen, Meghan E. Gallagher, MSc, Yuchen Ding, PhD, Sunita Goyal, MD, Clark Paramore, MPH, Alexis A. Thompson, MD, Mark C. Walters, MD

Blood (2020) 136 (Supplement 1): 10.

https://doi.org/10.1182/blood-2020-136193

Published: 2020

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

Resolution of Serious Vaso-Occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Alexis A. Thompson, MD, Mark C. Walters, MD, Markus Y Mapara, MD PhD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Banu Aygun, MD, Kimberly A. Kasow, DO, Stacey Rifkin-Zenenberg, DO, Manfred Schmidt, PhD, Jay DelCarpini, Francis J. Pierciey, Jr., Alexandra L. Miller, Meghan E. Gallagher, MSc, Ren Chen, Sunita Goyal, MD, Julie Kanter, MD, John F. Tisdale, MD

Blood (2020) 136 (Supplement 1): 16–17.

https://doi.org/10.1182/blood-2020-134940

Published: 2020

Journal Articles

Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease

Ruta Brazauskas, Graziana M. Scigliuolo, Hai-Lin Wang, Barbara Cappelli, Annalisa Ruggeri, Courtney D. Fitzhugh, Jane S. Hankins, Julie Kanter, Joerg J. Meerpohl, Julie A. Panepinto, Damiano Rondelli, Shalini Shenoy, Mark C. Walters, John E. Wagner, John F. Tisdale, Eliane Gluckman, Mary Eapen

Blood (2020) 136 (5): 623–626.

https://doi.org/10.1182/blood.2020005687

Published: 2020

Includes: Supplemental data

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

NARROW

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

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

The Impact of CD45-Antibody-Drug Conjugate Conditioning on Clonal Dynamics and Immune Tolerance Post HSPC Transplantation in Rhesus Macaques

Efficacy, Safety, and Health-Related Quality of Life (HRQOL) in Patients with Sickle Cell Disease (SCD) Who Have Received Lovotibeglogene Autotemcel (Lovo-cel) Gene Therapy: Up to 60 Months of Follow-up

Lovo-cel (bb1111) Gene Therapy for Sickle Cell Disease: Updated Clinical Results and Investigations into Two Cases of Anemia from Group C of the Phase 1/2 HGB-206 Study

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

Polyclonality Strongly Correlates with Biological Outcomes and Is Significantly Increased Following Improvements to the Phase 1/2 HGB-206 Protocol and Manufacturing of LentiGlobin for Sickle Cell Disease (SCD; bb1111) Gene Therapy (GT)

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

Sustained Improvements in Patient-Reported Quality of Life up to 24 Months Post-Treatment with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Gene therapy for sickle cell disease: moving from the bench to the bedside

Dietary iron restriction improves markers of disease severity in murine sickle cell anemia

Improvements in Health-Related Quality of Life for Patients Treated with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

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

Resolution of Serious Vaso-Occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease

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

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American Society of Hematology

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American Society of Hematology

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