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Journal Articles

Optimized Culture Medium for Enhanced Ex Vivo expansion of Human Hematopoietic Stem Cells

Janet J Sei, PhD, Blake S Moses, PhD, Abigail Harris-Becker, MS, MinJung Kim, PhD, Navjot Kaur, PhD, Mohan C Vemuri, PhD, Curt I Civin, MD

Blood (2019) 134 (Supplement_1): 1965.

https://doi.org/10.1182/blood-2019-126174

Published: 2019

Journal Articles

Ex Vivo Expansion with E478 Increases the Rate of CRISPR-Mediated Homology Directed Repair of the Beta-Globin Gene in Human Hematopoietic Stem Cells By >10-Fold and Improves In Vivo Engraftment By >120-Fold

Kevin A. Goncalves, PhD, Megan D. Hoban, PhD, Sharon L. Hyzy, MS, Katia S. George, BA, Anthony E. Boitano, PhD, Michael P. Cooke, PhD

Blood (2019) 134 (Supplement_1): 4634.

https://doi.org/10.1182/blood-2019-131157

Published: 2019

Journal Articles

EDIT-301: An Experimental Autologous Cell Therapy Comprising Cas12a-RNP Modified mPB-CD34+ Cells for the Potential Treatment of SCD

Edouard De Dreuzy, PhD, Jack Heath, John A Zuris, PhD, Patricia Sousa, Ramya Viswanathan, Sean Scott, Jen Da Silva, Terence Ta, PhD, Stacy Capehart, PhD, Tongyao Wang, Cecilia Fernandez, PhD, Vic E Myer, PhD, Charles F Albright, PhD, Christopher J Wilson, PhD, Sandra Teixeira, PhD, Kai-Hsin Chang, PhD

Blood (2019) 134 (Supplement_1): 4636.

https://doi.org/10.1182/blood-2019-130256

Published: 2019

Journal Articles

Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010

Blood (2019) 134 (Supplement_1): 3346.

https://doi.org/10.1182/blood-2019-124665

Published: 2019

Journal Articles

Dissecting Ex Vivo Expansion of Mobilized Peripheral Blood Hematopoietic Stem and Progenitor Cells By Single Cell RNA Sequencing

Matteo Maria Naldini, Gabriele Casirati, MD, Erika Zonari, PhD, Giacomo Desantis, PhD, Andrea Cammarata, PhD, Stefano Beretta, PhD, Ivan Merelli, PhD, Fabio Ciceri, MD, Bernhard Gentner, MD

Blood (2018) 132 (Supplement 1): 3343.

https://doi.org/10.1182/blood-2018-99-111879

Published: 2018

Journal Articles

MGTA-145 in Combination with Plerixafor Rapidly Mobilizes High Numbers of Hematopoietic Stem Cells and Graft-Versus-Host Disease Inhibiting Myeloid-Derived Suppressor Cells in Non-Human Primates

Kevin A. Goncalves, PhD, Patrick C. Falahee, PhD, Sharon L. Hyzy, MS, Shuping Li, PhD, Anthony E. Boitano, PhD, Dwight M. Morrow, PhD, Michael P. Cooke, PhD

Blood (2018) 132 (Supplement 1): 116.

https://doi.org/10.1182/blood-2018-99-117370

Published: 2018

Journal Articles

Infusion of a Cryopreservable Human Megakaryocyte-Biased Cell Product Results in Sustained Platelet Reconstitution In Vivo

Ami Patel, MS, Manisha Kintali, MS, Gohar Mosoyan, Ronald Hoffman, MD, Camelia Iancu-Rubin, PhD

Blood (2018) 132 (Supplement 1): 117.

https://doi.org/10.1182/blood-2018-99-118682

Published: 2018

Journal Articles

Protmune, a Next-Generation Graft for GvHD Prophylaxis in Allogeneic Hematopoietic Cell Transplantation: 1-Year Safety and Efficacy Phase 1 Data

Richard T. Maziarz, MD, John Edwards, MD, James Essell, MD, Cesar Freytes, MD, Chatchada Karanes, Ayman Saad, MD, Dimitrios Tzachanis, MD PhD, Monica Diaz, Amanda Medcalf, Chris Storgard, Abhinav Deol, MD

Blood (2018) 132 (Supplement 1): 2167.

https://doi.org/10.1182/blood-2018-99-115088

Published: 2018

Journal Articles

Comparative Studies Reveal Robust HbF Induction By Editing of HBG1/2 Promoters or BCL11A Erythroid-Enhancer in Human CD34+ Cells but That BCL11A Erythroid-Enhancer Editing Is Associated with Selective Reduction in Erythroid Lineage Reconstitution in a Xenotransplantation Model

Blood (2018) 132 (Supplement 1): 409.

https://doi.org/10.1182/blood-2018-99-114780

Published: 2018

Journal Articles

The Exhaustion of Adult Hematopoietic Stem Cells in Ex Vivo Cultures Can Be Overcome by a Histone Deacetylase Inhibitor

Eran Zimran, MD, Luena Papa, PhD, Mansour Djedaini, BS, Ronald Hoffman, MD

Blood (2017) 130 (Supplement 1): 655.

https://doi.org/10.1182/blood.V130.Suppl_1.655.655

Published: 2017

Journal Articles

Phenotype Does Not Always Equal Function: HDAC Inhibitors and UM171, but Not SR1, Lead to Rapid Upregulation of CD90 on Non-Engrafting CD34+CD90-Negative Human Cells

Kevin A. Goncalves, PhD, Megan D. Hoban, Jennifer L. Proctor, BS, Hillary L. Adams, MS, Sharon L. Hyzy, MS, Anthony E. Boitano, PhD, Michael P. Cooke, PhD

Blood (2017) 130 (Supplement 1): 659.

https://doi.org/10.1182/blood.V130.Suppl_1.659.659

Published: 2017

Journal Articles

Generation of Megakaryocytes and Platelets from Valproic Acid Expanded Human Cord Blood CD34+ Cells in Culture

Nadim Mahmud, MBBS, PhD, Rajeev Ranjan, PhD, Jia J. Wu, BS, Benjamin Petro, Meryem K. Terzioglu, MD, Hasan Issa, MBBch, Darryl A. Abbott, BS, Dolores Mahmud, PhD, William M. Miller, PhD

Blood (2017) 130 (Supplement 1): 3751.

https://doi.org/10.1182/blood.V130.Suppl_1.3751.3751

Published: 2017

Journal Articles

Efficient Ex Vivo Expansion of Human Umbilical Cord Blood-Derived Small CD133 + lin - CD45 - Stem Cells Expressing Pluripotency Markers in Presence of Histone Deacethylase Inhibitors—As a New Challenging Strategy to Obtain Multipotent Cells for Regenerative Medicine

Janina Ratajczak, MD PhD DSci, Malwina Suszynska, PhD, Kamila Bujko, MS, Donald M Miller, MD PhD, Magdalena Kucia, PhD DSci, Mariusz Z Ratajczak, MD PhD DSc

Blood (2017) 130 (Supplement 1): 3761.

https://doi.org/10.1182/blood.V130.Suppl_1.3761.3761

Published: 2017

Journal Articles

Expanding CRISPR Genome Editing Strategies in Hematopoietic Stem and Progenitor Cells for the Treatment of Hematologic Diseases

Edouard deDreuzy, PhD, Aditi Chalishazar, MS, Jack Heath, Carrie M Margulies, PhD, John Labella, Ramya Viswanathan, Katherine Loveluck, KaiHsin Chang, PhD, Hariharan Jayaram, PhD, John A Zuris, PhD, Vic E Myer, PhD, Charlie Albright, PhD, Cecilia Cotta-Ramusino, PhD, Jennifer L Gori, PhD

Blood (2017) 130 (Supplement 1): 4619.

https://doi.org/10.1182/blood.V130.Suppl_1.4619.4619

Published: 2017

Journal Articles

Pro-Tmune, the Next Generation Graft for Allogeneic Hematopoietic Cell Transplantation: Phase 1 Safety and Efficacy Data

Richard Maziarz, MD, John Edwards, MD, James Essell, MD, Cesar Freytes, MD, Chatchada Karanes, MD, Ayman Saad, MD, Dimitrios Tzachanis, MD PhD, Monica Diaz, Amanda Medcalf, Daniel Fremgen, Chris Storgard, MD, Abhinav Deol, MD

Blood (2017) 130 (Supplement 1): 4498.

https://doi.org/10.1182/blood.V130.Suppl_1.4498.4498

Published: 2017

Journal Articles

Aryl Hydrocarbon Receptor Antagonists Expand Adult Hematopoietic Stem Cells from Mobilized Peripheral Blood and Bone Marrow and Increase the Dose of CRISPR/Cas9 Gene-Edited NSG-Repopulating Cells

Megan D. Hoban, PhD, Kevin A. Goncalves, PhD, Jennifer L. Proctor, BS, Hillary L. Adams, MS, Sharon L. Hyzy, MS, Katia S. George, BA, Anthony E. Boitano, PhD, Michael P. Cooke, PhD

Blood (2017) 130 (Supplement 1): 3341.

https://doi.org/10.1182/blood.V130.Suppl_1.3341.3341

Published: 2017

Journal Articles

The Therapeutic Response of Platelet Rich Plasma (PRP) for Androgenetic Alopecia Showed No Correlation with Growth Factors and Platelet Number

Bruno Lima Rodrigues, Silmara Aparecida De Lima Montalvão, MSc, Joyce Annichinno-Bizzacchi, MD, Rebeca Cancela, MD, Francesca Silva, Aline Paffaro, Stephany Cares Huber, MSc, José Luiz Rosenberis Cunha Junior, MSc, Erich Vinicius De Paula, MD PhD

Blood (2016) 128 (22): 2637.

https://doi.org/10.1182/blood.V128.22.2637.2637

Published: 2016

Journal Articles

Incremental Innovation of Ex Vivo Hematopoietic Stem Cell Engineering to Expand Clinical Gene Therapy Applications

Erika Zonari, PhD, Giacomo Desantis, PhD, Carolina Petrillo, Oriana Meo, Samantha Scaramuzza, PhD, Maria Rosa Lidonnici, PhD, Anna Kajaste-Rudnitski, PhD, Fabio Ciceri, MD, Sarah Marktel, Alessandro Aiuti, MD PhD, Giuliana Ferrari, PhD, Luigi Naldini, MD PhD, Bernhard Gentner, MD

Blood (2016) 128 (22): 4707.

https://doi.org/10.1182/blood.V128.22.4707.4707

Published: 2016

Journal Articles

In Vitro Expanded γδ T Cells Derived from Cord Blood Induce Potent and Specific Cytotoxicity Against Human Acute Myeloid Leukemia Cells

Alice MS Cheung, PhD, Kar Wai Tan, PhD, Dian Yan Guo, BSc, Su-Ann Goh, BSc, Amanda SY Lau, BSc, Yeh Ching Linn, MBBS, MRCP, William YK Hwang, MBBS, MRCP, FRCP, Yeow Tee Goh, MD, Shang Li, MD PhD

Blood (2016) 128 (22): 2168.

https://doi.org/10.1182/blood.V128.22.2168.2168

Published: 2016

Journal Articles

Preclinical Development and Validation of a Hematopoietic Stem Cell-Based Lentivector Treatment for Hemophilia a

Christopher B Doering, PhD, Gabriela Denning, PhD, Jordan E Shields, MS, William Swaney, Lilith Reeves, Punam Malik, MD, H. Trent Spencer, PhD

Blood (2015) 126 (23): 4421.

https://doi.org/10.1182/blood.V126.23.4421.4421

Published: 2015

NARROW

Optimized Culture Medium for Enhanced Ex Vivo expansion of Human Hematopoietic Stem Cells

Ex Vivo Expansion with E478 Increases the Rate of CRISPR-Mediated Homology Directed Repair of the Beta-Globin Gene in Human Hematopoietic Stem Cells By >10-Fold and Improves In Vivo Engraftment By >120-Fold

EDIT-301: An Experimental Autologous Cell Therapy Comprising Cas12a-RNP Modified mPB-CD34+ Cells for the Potential Treatment of SCD

Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010

Dissecting Ex Vivo Expansion of Mobilized Peripheral Blood Hematopoietic Stem and Progenitor Cells By Single Cell RNA Sequencing

MGTA-145 in Combination with Plerixafor Rapidly Mobilizes High Numbers of Hematopoietic Stem Cells and Graft-Versus-Host Disease Inhibiting Myeloid-Derived Suppressor Cells in Non-Human Primates

Infusion of a Cryopreservable Human Megakaryocyte-Biased Cell Product Results in Sustained Platelet Reconstitution In Vivo

Protmune, a Next-Generation Graft for GvHD Prophylaxis in Allogeneic Hematopoietic Cell Transplantation: 1-Year Safety and Efficacy Phase 1 Data

Comparative Studies Reveal Robust HbF Induction By Editing of HBG1/2 Promoters or BCL11A Erythroid-Enhancer in Human CD34+ Cells but That BCL11A Erythroid-Enhancer Editing Is Associated with Selective Reduction in Erythroid Lineage Reconstitution in a Xenotransplantation Model

The Exhaustion of Adult Hematopoietic Stem Cells in Ex Vivo Cultures Can Be Overcome by a Histone Deacetylase Inhibitor

Phenotype Does Not Always Equal Function: HDAC Inhibitors and UM171, but Not SR1, Lead to Rapid Upregulation of CD90 on Non-Engrafting CD34+CD90-Negative Human Cells

Generation of Megakaryocytes and Platelets from Valproic Acid Expanded Human Cord Blood CD34+ Cells in Culture

Efficient Ex Vivo Expansion of Human Umbilical Cord Blood-Derived Small CD133 + lin - CD45 - Stem Cells Expressing Pluripotency Markers in Presence of Histone Deacethylase Inhibitors—As a New Challenging Strategy to Obtain Multipotent Cells for Regenerative Medicine

Expanding CRISPR Genome Editing Strategies in Hematopoietic Stem and Progenitor Cells for the Treatment of Hematologic Diseases

Pro-Tmune, the Next Generation Graft for Allogeneic Hematopoietic Cell Transplantation: Phase 1 Safety and Efficacy Data

Aryl Hydrocarbon Receptor Antagonists Expand Adult Hematopoietic Stem Cells from Mobilized Peripheral Blood and Bone Marrow and Increase the Dose of CRISPR/Cas9 Gene-Edited NSG-Repopulating Cells

The Therapeutic Response of Platelet Rich Plasma (PRP) for Androgenetic Alopecia Showed No Correlation with Growth Factors and Platelet Number

Incremental Innovation of Ex Vivo Hematopoietic Stem Cell Engineering to Expand Clinical Gene Therapy Applications

In Vitro Expanded γδ T Cells Derived from Cord Blood Induce Potent and Specific Cytotoxicity Against Human Acute Myeloid Leukemia Cells

Preclinical Development and Validation of a Hematopoietic Stem Cell-Based Lentivector Treatment for Hemophilia a

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American Society of Hematology

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