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Journal Articles

Exploring the Drivers of Potential Clinical Benefit in Initial Patients Treated in the Hgb-206 Study of Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy

Mark C. Walters, MD, John F. Tisdale, MD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Markus Y. Mapara, MD PhD, Manfred Schmidt, PhD, Alexandra L. Miller, Francis J. Pierciey, Jr., Wenmei Huang, PhD MS, Jean-Antoine Ribeil, MD PhD, Julie Kanter, MD, Alexis A. Thompson, MD MPH

Blood (2019) 134 (Supplement_1): 2061.

https://doi.org/10.1182/blood-2019-128814

Published: 2019

Journal Articles

Northstar-2: Updated Safety and Efficacy Analysis of Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Non-β0/β0 Genotypes

Alexis A. Thompson, MD MPH, Mark C. Walters, MD, Janet L. Kwiatkowski, MD MSCE, Suradej Hongeng, MD, John B. Porter, MA MDFRCP FRCPath, Martin G. Sauer, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, Isabelle Thuret, MD, Heidi Elliot, Ge Tao, PhD, Richard A. Colvin, MD PhD, Franco Locatelli, MD PhD

Blood (2019) 134 (Supplement_1): 3543.

https://doi.org/10.1182/blood-2019-126046

Published: 2019

Journal Articles

Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

Angela R. Smith, MD MS, Gary J. Schiller, MD, Gregory M Vercellotti, MD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Erica B. Esrick, MD, David A. Williams, MD, Weston P. Miller, MD, Adrian Woolfson, MD PhD, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 3544.

https://doi.org/10.1182/blood-2019-125743

Published: 2019

Journal Articles

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

Janet L. Kwiatkowski, MD MSCE, Alexis A. Thompson, MD MPH, John E.J. Rasko, MBBS, PhD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, MD, Marina Cavazzana, MD PhD, P. Joy Ho, MBBS DPhil FRACP FRCPA, Manfred Schmidt, PhD, Morris Kletzel, MD MBA, Elliott P. Vichinsky, MD, Briana Deary, Ying Chen, PhD, Alexandria Petrusich, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 4628.

https://doi.org/10.1182/blood-2019-125807

Published: 2019

Journal Articles

Genome Wide Interaction Analysis Identifies Expression Quantitative Trait Loci Associated with Reduced Survival after Reduced Intensity Conditioning HLA-Matched Unrelated Donor Allogeneic Hematopoietic Cell Transplant

Blood (2019) 134 (Supplement_1): 4595.

https://doi.org/10.1182/blood-2019-129182

Published: 2019

Journal Articles

Role of B-Cell Maturation Antigen (BCMA) Targeted Immunotherapies in Relapsed and Refractory Multiple Myeloma- a Systematic Review

Zunairah Shah, MBBS, Syeda Sabeeka Batool, MBBS, MD, Hafiz Muhammad Fazeel, MBBS, Mustafa Nadeem Malik, MD, Maryam Sadiq, MBBS, Syeda Fatima Hassan, MBBS, Tariq Iqtidar Sadiq Syed, MD, Syed Maaz Abdullah, MBBS, Nisma Javed, MBBS, Aqsa Ashraf, MD, Arafat Ali Ali Farooqui, MD, Muhammad Abdullah Yousaf, MBBS, MD, Muhaddis Ejaz Ahmad, MD, Marium Ghani, MBBS, Arshia Akbar, MD, Faiz Anwer, MD

Blood (2019) 134 (Supplement_1): 5597.

https://doi.org/10.1182/blood-2019-131849

Published: 2019

Journal Articles

A Novel Targeted Approach to Achieve Immune System Reset: CD45-Targeted Antibody Drug Conjugates Enable Autologous HSCT, Ameliorate Disease in Autoimmune Models, Potently Kill Human Immune Cells from Normal Donors and MS Patients, and Achieve Immune Depletion in Non-Human Primates (NHP)

Geoff O Gillard, Jennifer L Proctor, BS, Melissa L. Brooks, BS, Tahirih L Lamothe, BS, Sharon L. Hyzy, MS, Oliver Mikse, Sean McDonough, Rahul Palchaudhuri, PhD, Anjali Bhat, MS, Ganapathy N. Sarma, PhD, Prashant Bhattarai, Pranoti Sawant, MS, Bradley R Pearse, PhD, Charlotte F. Mcdonough, PhD, Anthony E. Boitano, PhD, Michael P. Cooke, PhD

Blood (2019) 134 (Supplement_1): 3208.

https://doi.org/10.1182/blood-2019-130752

Published: 2019

Journal Articles

Use of Ex Vivo Graft Manipulation and Posttransplant Cyclophosphamide Result in Low GvHD Rates and Acceptable Engraftment after RIC Regimens in Pediatric Mismatched SCT

Peter Lang, MD, Michaela Döring, PD Dr. MD, Anne-Marie Lang, M.Sc., Patrick Schlegel, Dr. MD, Christian M. Seitz, Dr. MD, Robin Fischer, Dr. rer. nat., Ursula Holzer, PD Dr. MD, Ines Brecht, PD Dr. MD, Rupert Handgretinger, MD

Blood (2019) 134 (Supplement_1): 3255.

https://doi.org/10.1182/blood-2019-129654

Published: 2019

Journal Articles

GATA2 -Dependent Developmental and Regenerative Networks

Alexandra Soukup, PhD, Kirby D Johnson, PhD, Daniel J Conn, PhD, Evgenia Shishkova, PhD, Koichi Ricardo Katsumura, MD PhD, Peng Liu, PhD, Erik A. Ranheim, MD PhD, Katherine R. Calvo, MD PhD, Amy P. Hsu, Steven M. Holland, MD, Joshua J Coon, PhD, Sunduz Keles, PhD, Emery H Bresnick, PhD

Blood (2019) 134 (Supplement_1): 1182.

https://doi.org/10.1182/blood-2019-126875

Published: 2019

Journal Articles

Local Irradiation Induces Systemic Inflammatory Response and Alteration of the Hematopoietic Stem Cell Niche

Yuko Kawano, MD PhD, Daniel K Byun, BS, Benjamin J. Frisch, PhD, Hiroki Kawano, MD PhD, Mark W LaMere, BA, Carl J. Johnston, PhD, Jaqueline P. Williams, PhD, Laura M. Calvi, MD

Blood (2019) 134 (Supplement_1): 1213.

https://doi.org/10.1182/blood-2019-131809

Published: 2019

Journal Articles

Aging of Bone Marrow Microenvironment Promotes Myeloid Bias of Hematopoietic Progenitors and Is a Target in Age-Related Myeloproliferative Neoplasms

Blood (2018) 132 (Supplement 1): 3842.

https://doi.org/10.1182/blood-2018-99-116015

Published: 2018

Journal Articles

Granulopoiesis in the Control of Hematopoietic Stem Cell Self-Renewal and Niches

Daniel Lucas-Alcaraz, PhD

Blood (2018) 132 (Supplement 1): SCI-21.

https://doi.org/10.1182/blood-2018-99-109517

Published: 2018

Journal Articles

Endothelial MAPK Activation Disrupts Hematopoiesis By Depleting Plasma SCGF

Jason M. Butler, PhD, Pradeep Ramalingam, MD, Elisa Lazzari, PhD, Michael Poulos, PhD

Blood (2018) 132 (Supplement 1): 1290.

https://doi.org/10.1182/blood-2018-99-117467

Published: 2018

Journal Articles

Current Results of Lentiglobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under a Refined Protocol in the Phase 1 Hgb-206 Study

John F. Tisdale, MD, Julie Kanter, MD, Markus Y. Mapara, MD PhD, Janet L. Kwiatkowski, MD, Lakshmanan Krishnamurti, MD, Manfred Schmidt, PhD, Alexandra L. Miller, Francis J. Pierciey, Jr., Weiliang Shi, PhD, Jean-Antoine Ribeil, MD PhD, Mohammed Asmal, MD PhD, Alexis A. Thompson, MD MPH, Mark C. Walters, MD

Blood (2018) 132 (Supplement 1): 1026.

https://doi.org/10.1182/blood-2018-99-113480

Published: 2018

Journal Articles

Diversity and Richness Analysis of the Oral and Gastrointestinal Microbiome during Autologous Transplantation for Multiple Myeloma: Results of a Prospective Pilot Study and Correlation with Transplant Outcomes

Najla H El Jurdi, MD, Ali Filali, PhD, Iman Salem, MD, Mauricio Retuerto, MS, Nina Dambrosio, CNP, Hillard M Lazarus, MD, Paolo F Caimi, MD, Richard Creger, PharmD, Brenda Cooper, MD, Ben Tomlinson, MD, Leland Metheny, MD, Ehsan Malek, MD, Folashade Otegbeye, MBBChir, MPH, Rafick-Pierre Sekaly, PhD, Mahmoud Ghannoum, PhD, Marcos de Lima, MD

Blood (2018) 132 (Supplement 1): 4627.

https://doi.org/10.1182/blood-2018-99-120038

Published: 2018

Journal Articles

Improved Relapse Rate, T Cell Recovery and Engraftment in High Risk Hematological Malignancies with Enhanced Lympho-Depletion Prior to T-Cell Replete Peripheral Blood Haploidentical (HID) Hematopoietic Stem Cell Transplantation (HCT)

Yasser Khaled, MD, Joshua Boss, RN, OCN, BMTCN, Joel Gradowski, MD, Robert Bradley, MD, Jason C. Chandler, MD

Blood (2018) 132 (Supplement 1): 2117.

https://doi.org/10.1182/blood-2018-99-115290

Published: 2018

Journal Articles

Estrogens Enhance Human Hematopoietic Stem Cell Engraftment in a Xenogenic Transplantation Model

Sara Fañanas-Baquero, Israel Orman, Federico Becerra Aparicio, Silvia Bermudez de Miguel, Jordi Garcia Merino, Rosa M Yañez, PhD, Yolanda Fernandez, David Alfaro, Agustin Zapata, Juan A. Bueren, PhD, Jose C Segovia, PhD, Oscar Quintana Bustamante, PhD

Blood (2018) 132 (Supplement 1): 2042.

https://doi.org/10.1182/blood-2018-99-112104

Published: 2018

Journal Articles

Cord Blood Units (CBU) Selected for Transplantation from a Single CB Bank over 10 Years: High Correlation of Potency/Quality Among Pre-Cryopreservation and Post-Thaw Segment Measurements and Time to Engraftment

Maria S. Albano, PhD, Ludy Dobrila, PhD, Michal Tarnawski, MD, Dorothy Sung, Emeline Masson Frenet, PhD, Cynthia Romeo, Tracy Zhu, Chiseko Watanabe, Dan Zamfir, Siddarth Krishnan, Pablo Rubinstein, MD, Andromachi Scaradavou, MD

Blood (2018) 132 (Supplement 1): 2062.

https://doi.org/10.1182/blood-2018-99-116514

Published: 2018

Journal Articles

Thalassemia Gene Therapy By In Vivo Transduction of Mobilized Hematopoietic Stem Cells (HSCs) with an Integrating Hybrid Adenovirus Vector System

Afrodite Georgakopoulou, PhDstudent, Hongjie Wang, PhD, Chrysi Kapsali, Undergraduate, Nikoleta Psatha, PhD, Angeliki Koufali, Graduate, Chang Li, PhD, Zsuzsanna Izsvak, PhD, Achilles Anagnostopoulos, Thalia Papayannopoulou, MD DrSci, Evangelia Yannaki, MD, Andre Lieber, MD PhD

Blood (2018) 132 (Supplement 1): 2193.

https://doi.org/10.1182/blood-2018-99-116787

Published: 2018

Journal Articles

Valine as a Key Metabolic Regulator of Hematopoietic Stem Cell Maintenance

Hiromitsu Nakauchi, MD PhD

Blood (2017) 130 (Supplement 1): SCI-20.

https://doi.org/10.1182/blood.V130.Suppl_1.SCI-20.SCI-20

Published: 2017

NARROW

Exploring the Drivers of Potential Clinical Benefit in Initial Patients Treated in the Hgb-206 Study of Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy

Northstar-2: Updated Safety and Efficacy Analysis of Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Non-β0/β0 Genotypes

Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

Genome Wide Interaction Analysis Identifies Expression Quantitative Trait Loci Associated with Reduced Survival after Reduced Intensity Conditioning HLA-Matched Unrelated Donor Allogeneic Hematopoietic Cell Transplant

Role of B-Cell Maturation Antigen (BCMA) Targeted Immunotherapies in Relapsed and Refractory Multiple Myeloma- a Systematic Review

A Novel Targeted Approach to Achieve Immune System Reset: CD45-Targeted Antibody Drug Conjugates Enable Autologous HSCT, Ameliorate Disease in Autoimmune Models, Potently Kill Human Immune Cells from Normal Donors and MS Patients, and Achieve Immune Depletion in Non-Human Primates (NHP)

Use of Ex Vivo Graft Manipulation and Posttransplant Cyclophosphamide Result in Low GvHD Rates and Acceptable Engraftment after RIC Regimens in Pediatric Mismatched SCT

GATA2 -Dependent Developmental and Regenerative Networks

Local Irradiation Induces Systemic Inflammatory Response and Alteration of the Hematopoietic Stem Cell Niche

Aging of Bone Marrow Microenvironment Promotes Myeloid Bias of Hematopoietic Progenitors and Is a Target in Age-Related Myeloproliferative Neoplasms

Granulopoiesis in the Control of Hematopoietic Stem Cell Self-Renewal and Niches

Endothelial MAPK Activation Disrupts Hematopoiesis By Depleting Plasma SCGF

Current Results of Lentiglobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under a Refined Protocol in the Phase 1 Hgb-206 Study

Diversity and Richness Analysis of the Oral and Gastrointestinal Microbiome during Autologous Transplantation for Multiple Myeloma: Results of a Prospective Pilot Study and Correlation with Transplant Outcomes

Improved Relapse Rate, T Cell Recovery and Engraftment in High Risk Hematological Malignancies with Enhanced Lympho-Depletion Prior to T-Cell Replete Peripheral Blood Haploidentical (HID) Hematopoietic Stem Cell Transplantation (HCT)

Estrogens Enhance Human Hematopoietic Stem Cell Engraftment in a Xenogenic Transplantation Model

Cord Blood Units (CBU) Selected for Transplantation from a Single CB Bank over 10 Years: High Correlation of Potency/Quality Among Pre-Cryopreservation and Post-Thaw Segment Measurements and Time to Engraftment

Thalassemia Gene Therapy By In Vivo Transduction of Mobilized Hematopoietic Stem Cells (HSCs) with an Integrating Hybrid Adenovirus Vector System

Valine as a Key Metabolic Regulator of Hematopoietic Stem Cell Maintenance

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American Society of Hematology

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