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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

Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua

Nisha Nair, Dries De Wolf, Phuong Anh Nguyen, Quang Hong Pham, Ermira Samara-Kuko, Jeff Landau, Grant E. Blouse, Marinee K. Chuah, Thierry VandenDriessche

Blood (2021) 137 (21): 2902–2906.

https://doi.org/10.1182/blood.2020006005

Published: 2021

Includes: Supplemental data

Journal Articles

No CpGs for AAVs?

Lindsey A. George

Blood (2021) 137 (6): 721–723.

https://doi.org/10.1182/blood.2020009285

Published: 2021

Journal Articles

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Barbara A. Konkle, Christopher E. Walsh, Miguel A. Escobar, Neil C. Josephson, Guy Young, Annette von Drygalski, Scott W. J. McPhee, R. Jude Samulski, Ivan Bilic, Maurus de la Rosa, Birgit M. Reipert, Hanspeter Rottensteiner, Friedrich Scheiflinger, John C. Chapin, Bruce Ewenstein, Paul E. Monahan

Blood (2021) 137 (6): 763–774.

https://doi.org/10.1182/blood.2019004625

Published: 2021

Includes: Supplemental data

Journal Articles

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

Blood (2020) 135 (15): 1219–1231.

https://doi.org/10.1182/blood.2019002350

Published: 2020

Includes: Supplemental data

Journal Articles

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Erica B. Esrick, MD, Maureen Achebe, MD MPH, Myriam Armant, PhD, Pablo Bartolucci, MD PhD, Marioara Felicia Ciuculescu, MD, Heather Daley, Colleen Dansereau, MSN, RN, CPN, Giuseppe Di Caprio, PhD, Bronner Goncalves, MD PhD, Nicolas Hebert, Matthew M Heeney, MD, John M. Higgins, MD PhD, Leslie E. Lehmann, MD, John P Manis, MD, Olivier Negre, PhD, Sarah Nikiforow, MD PhD, Ethan Schonbrun, PhD, David K. Wood, PhD, David A. Williams, MD

Blood (2019) 134 (Supplement_2): LBA-5.

https://doi.org/10.1182/blood-2019-132745

Published: 2019

Journal Articles

Aberrant Cathepsin S Induces a Supportive Immune Microenvironment in Follicular Lymphoma

Blood (2019) 134 (Supplement_1): 657.

https://doi.org/10.1182/blood-2019-122663

Published: 2019

Journal Articles

Gata2 Expression and Function in PML-RARA- driven Preleukemia and APL

Casey D.S. Katerndahl, PhD, Timothy J Ley, MD, Michelle A Cai, Timothy P Rooney, Jr., Sai Mukund Ramakrishnan, MS, Sridhar Nonavinkere Srivatsan, MS, Christopher A Miller, PhD

Blood (2019) 134 (Supplement_1): 534.

https://doi.org/10.1182/blood-2019-131883

Published: 2019

Journal Articles

Germline RUNX1 Variation and Predisposition to T-Cell Acute Lymphoblastic Leukemia in Children

Blood (2019) 134 (Supplement_1): 653.

https://doi.org/10.1182/blood-2019-125912

Published: 2019

Journal Articles

Mutations and Copy Number Gains of the BCL2 Family Members Mediate Resistance to Venetoclax in Multiple Myeloma (MM) Patients

Paola Neri, MD PhD, Ranjan Maity, PhD, Jean-Baptiste Alberge, Sarthak Sinha, BSc, Justin Donovan, Madison Kong, Fajer Hasan, Arzina Jaffer, Lawrence H. Boise, PhD, Nizar Bahlis, MD

Blood (2019) 134 (Supplement_1): 572.

https://doi.org/10.1182/blood-2019-127593

Published: 2019

Journal Articles

Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial

Bernhard Gentner, MD, Maria Ester Bernardo, MD PhD, Francesca Tucci, MD, Erika Zonari, PhD, Francesca Fumagalli, MD, Silvia Pontesilli, MD, Serena Acquati, PhD, Paolo Silvani, MD, Fabio Ciceri, MD, Attilio Rovelli, MD, Giancarlo La Marca, PhD, Rossella Parini, MD, Silvia Gregori, PhD, Eugenio Montini, PhD, Luigi Naldini, MD PhD, Alessandro Aiuti, MD PhD

Blood (2019) 134 (Supplement_1): 607.

https://doi.org/10.1182/blood-2019-128805

Published: 2019

Journal Articles

Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined Immunodeficiency

Blood (2019) 134 (Supplement_1): 608.

https://doi.org/10.1182/blood-2019-127439

Published: 2019

Journal Articles

Long-Term AAV-Mediated Factor VIII Expression in Nine Hemophilia A Dogs: A 10 Year Follow-up Analysis on Durability, Safety and Vector Integration

Giang N. Nguyen, BS, John K. Everett, PhD, Hayley Raymond, BA, Samita Kafle, BS, Elizabeth P. Merricks, PhD, Haig H. Kazazian, MD, Timothy C. Nichols, MD, Frederic D. Bushman, PhD, Denise E. Sabatino, PhD

Blood (2019) 134 (Supplement_1): 611.

https://doi.org/10.1182/blood-2019-126007

Published: 2019

Journal Articles

The Immunophilin FKBP12 Inhibits Hepcidin By Modulating BMP Type I-Type II Receptors Interaction and Ligand Responsiveness

Alessandro Dulja, MSc, Alessia Pagani, PhD, Mariateresa Pettinato, Antonella Nai, PhD, Clara Camaschella, MD, Laura Silvestri, PhD

Blood (2019) 134 (Supplement_1): 430.

https://doi.org/10.1182/blood-2019-130058

Published: 2019

Journal Articles

HDAC8 Promotes MLL-Rearranged Acute Myeloid Leukemia By Inhibiting p53 Acetylation

Dijiong Wu, MD, Man Li, PhD, Lianjun Zhang, PhD, Jing Qi, PhD, Chun-Wei David Chen, PhD, Xin He, PhD, Yinghui Zhu, PhD, Haojie Dong, PhD, Jie Sun, PhD, Ling Li, PhD, Guido Marcucci, MD, Ya-Huei Kuo, PhD

Blood (2019) 134 (Supplement_1): 642.

https://doi.org/10.1182/blood-2019-124134

Published: 2019

Journal Articles

Effective Stabilization of Viral Vectors in Liquid Using an Algorithm-Based Development Approach

Konstantin Petropoulos, PhD

Blood (2019) 134 (Supplement_1): 5752.

https://doi.org/10.1182/blood-2019-130030

Published: 2019

Journal Articles

Optimal Dual-Targeted CAR Construct Simultaneously Targeting Bcma and GPRC5D Prevents Bcma-Escape Driven Relapse in Multiple Myeloma

Carlos Fernandez de Larrea, MD PhD, Mette Staehr, PhD, Andrea Lopez, Yunxin Chen, MBBS, Terence J Purdon, MS, Khong Y. Ng, PhD, Hans Wendel, MD, Renier J. Brentjens, MD PhD, Eric L Smith, MD PhD

Blood (2019) 134 (Supplement_1): 136.

https://doi.org/10.1182/blood-2019-126145

Published: 2019

Journal Articles

The Relationships between Target Gene Transduction, Engraftment of HSCs and RBC Physiology in Sickle Cell Disease Gene Therapy

Melissa Bonner, PhD, Julie Kanter, MD, Elizabeth Macari, PhD, Ricky Lane, Gretchen Lewis, BMBS, Paige Coles, Sarah Kassenaar, Sai Mynampati, MS, Robert Schulze, Madison Hebert, Mark C. Walters, MD, Alexis A. Thompson, MD MPH, Mohammed Asmal, MD PhD, John F. Tisdale, MD, Francis J. Pierciey, Jr.

Blood (2019) 134 (Supplement_1): 206.

https://doi.org/10.1182/blood-2019-129124

Published: 2019

Journal Articles

VAV1 Mutations Contributes to the Development of T-Cell Malignancies in Mice

Kota Fukumoto, MD, Mamiko Sakata-Yanagimoto, MD PhD, Manabu Fujisawa, MD, Tatsuhiro Sakamoto, MD PhD, Tran B. Nguyen, Sakurako Suma, MD, Hiroaki Miyoshi, MD PhD, Koichi Ohshima, MD PhD, Keisuke Kataoka, MD PhD, Seishi Ogawa, MD PhD, Shigeru Chiba, MD PhD

Blood (2019) 134 (Supplement_1): 300.

https://doi.org/10.1182/blood-2019-124591

Published: 2019

Journal Articles

Single Cell RNA Sequencing Identifies Transcriptional Programs That Enhance Anti-Tumor Function of Transgenic CD4+ T Cells Redirected with TCR and CD8αβ

Jan A Rath, MS, Gagan Bajwa, PhD, Benoit Carreres, MS, Isabelle Gruber, MS, Elisabeth Hoyer, BS, Meng-Fen Wu, MS, Yi-Ru Yu, PhD, Ping-Chih Ho, PhD, Malcolm K. Brenner, MD PhD, David Gfeller, PhD, Caroline Arber, MD

Blood (2019) 134 (Supplement_1): 250.

https://doi.org/10.1182/blood-2019-124714

Published: 2019

NARROW

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

Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua

No CpGs for AAVs?

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Aberrant Cathepsin S Induces a Supportive Immune Microenvironment in Follicular Lymphoma

Gata2 Expression and Function in PML-RARA- driven Preleukemia and APL

Germline RUNX1 Variation and Predisposition to T-Cell Acute Lymphoblastic Leukemia in Children

Mutations and Copy Number Gains of the BCL2 Family Members Mediate Resistance to Venetoclax in Multiple Myeloma (MM) Patients

Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial

Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined Immunodeficiency

Long-Term AAV-Mediated Factor VIII Expression in Nine Hemophilia A Dogs: A 10 Year Follow-up Analysis on Durability, Safety and Vector Integration

The Immunophilin FKBP12 Inhibits Hepcidin By Modulating BMP Type I-Type II Receptors Interaction and Ligand Responsiveness

HDAC8 Promotes MLL-Rearranged Acute Myeloid Leukemia By Inhibiting p53 Acetylation

Effective Stabilization of Viral Vectors in Liquid Using an Algorithm-Based Development Approach

Optimal Dual-Targeted CAR Construct Simultaneously Targeting Bcma and GPRC5D Prevents Bcma-Escape Driven Relapse in Multiple Myeloma

The Relationships between Target Gene Transduction, Engraftment of HSCs and RBC Physiology in Sickle Cell Disease Gene Therapy

VAV1 Mutations Contributes to the Development of T-Cell Malignancies in Mice

Single Cell RNA Sequencing Identifies Transcriptional Programs That Enhance Anti-Tumor Function of Transgenic CD4+ T Cells Redirected with TCR and CD8αβ

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American Society of Hematology

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