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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

Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

Laura Breda, PhD, Valentina Ghiaccio, PhD, Silvia Pires Lourenco, MSc, Danuta Jadwiga Jarocha, PhD, Yasuhiro Ikawa, MD PhD, Naoto Tanaka, PhD, Virginia Guzikowski, Carla Casu, PhD, Kim Smith-Whitley, MD, Janet L. Kwiatkowski, MD MSCE, Stefano Rivella, PhD

Blood (2019) 134 (Supplement_1): 2242.

https://doi.org/10.1182/blood-2019-124043

Published: 2019

Journal Articles

Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Blood (2019) 134 (Supplement_1): 4629.

https://doi.org/10.1182/blood-2019-126161

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

First-in-human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A

Steven Pipe, MD, Michael Becka, Elke Detering, Konstantina Vanevski, Toshko Lissitchkov

Blood (2019) 134 (Supplement_1): 4630.

https://doi.org/10.1182/blood-2019-125764

Published: 2019

Journal Articles

Diplotype Analysis of NUDT15 Variants By Targeted Sequencing in Pediatric Acute Lymphoblastic Leukemia

Chih-Hsiang Yu, Ya-Hsuan Chang, PhD, Wen-Chien Chou, MD, PhD, Der-Shiun Wang, MD, Shu-Wha Lin, PhD, Hsuan-Yu Chen, PhD, Yung-Li Yang, MD PhD

Blood (2019) 134 (Supplement_1): 1305.

https://doi.org/10.1182/blood-2019-124053

Published: 2019

Journal Articles

Control of HIV-1 Infection By Ferroportin Q248H Mutation

Namita Kumari, PhD, Seyed Mehdi Nouraie, PhD MD, Hatajai Lassiter, MS, BS, Asrar Ahmad, PhD, Kathryn Anastos, Jason Lazar, Seble Kassaye, Bradley Aouizerat, Audrey French, Stephen Gange, Adaora Adimora, Neale Weitzmann, Margaret Fischl, Mirjam-Colette Kempf, Sergei Nekhai, PhD

Blood (2019) 134 (Supplement_1): 953.

https://doi.org/10.1182/blood-2019-128685

Published: 2019

Journal Articles

Longitudinal Tracking of Acute Myeloid Leukemia Stem Cells in Xenografts and Patients By microRNA Reporters and Single Cell RNA Sequencing

Matteo Maria Naldini, Gabriele Casirati, MD, Matteo Barcella, PhD, Stefano Beretta, PhD, Ivan Merelli, PhD, Carolina Caserta, Giacomo Desantis, PhD, Francesca Pavesi, MD, Fabio Ciceri, MD, Bernhard Gentner, MD

Blood (2019) 134 (Supplement_1): 916.

https://doi.org/10.1182/blood-2019-131167

Published: 2019

Journal Articles

Machine Learning Algorithms in Predicting Hospital Readmissions in Sickle Cell Disease

Arisha Patel, MD, Kyra Gan, PhDCandidate, Andrew Li, PhD, Jeremy Weiss, MD PhD, Seyed Mehdi Nouraie, MD PhD, Sridhar Tayur, PhD, Enrico M Novelli, MD MS

Blood (2019) 134 (Supplement_1): 982.

https://doi.org/10.1182/blood-2019-121808

Published: 2019

Journal Articles

Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study

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

Blood (2019) 134 (Supplement_1): 990.

https://doi.org/10.1182/blood-2019-128894

Published: 2019

Journal Articles

Academic, Phase I/II Trial on T Cells Expressing a Second Generation, CD19-Specific Chimeric Antigen Receptor (CAR) and Inducible Caspase 9 Safety Switch for the Treatment of B-Cell Precursor Acute Lymphoblastic Leukemia (BCP-ALL) and B-Cell Non-Hodgkin Lymphoma (B-NHL) in Children

Francesca Del Bufalo, MD, Concetta Quintarelli, PhD, Biagio De Angelis, PhD, Ignazio Caruana, PhD, Matilde Sinibaldi, PhD, Luciana Vinti, MD, Pietro Merli, MD, Mattia Algeri, MD, Annalisa Ruggeri, MD, Federica Galaverna, MD, Valentina Cirillo, PhD, Maria Giuseppina Cefalo, MD, Giuseppina Li Pira, PhD, Giovanna Leone, MD, Valentina Bertaina, PhD, Franca Fassio, PhD, Monica Gunetti, PhD, Stefano Iacovelli, PhD, Franco Locatelli, MD PhD

Blood (2019) 134 (Supplement_1): 1341.

https://doi.org/10.1182/blood-2019-129821

Published: 2019

Journal Articles

Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene

Ashutosh Lal, MD, Franco Locatelli, MD PhD, Janet L. Kwiatkowski, MD MSCE, Andreas E. Kulozik, MD PhD, Evangelia Yannaki, MD, John B. Porter, MA MDFRCP FRCPath, Isabelle Thuret, MD, Martin G. Sauer, MD, Heidi Elliot, Ying Chen, PhD, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD MPH

Blood (2019) 134 (Supplement_1): 815.

https://doi.org/10.1182/blood-2019-128482

Published: 2019

Journal Articles

Expression Profiling of mRNA By Next Generation Sequencing and the Development of Algorithm for Predicting Response in Acute Myeloid Leukemia

Maher Albitar, MD, Marina Y Konopleva, MD PhD, Hong Zhang, PhD, Ivan De Dios, BS, Wanlong Ma, Spiraggelos Antzoulatos, Steven M. Kornblau, MD, Antonio Cavazos, Sanam Loghavi, MD, Koichi Takahashi, MD, Hagop M. Kantarjian, MD, Courtney D. DiNardo, MDMSc

Blood (2019) 134 (Supplement_1): 1314.

https://doi.org/10.1182/blood-2019-127449

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

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

Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

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

First-in-human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A

Diplotype Analysis of NUDT15 Variants By Targeted Sequencing in Pediatric Acute Lymphoblastic Leukemia

Control of HIV-1 Infection By Ferroportin Q248H Mutation

Longitudinal Tracking of Acute Myeloid Leukemia Stem Cells in Xenografts and Patients By microRNA Reporters and Single Cell RNA Sequencing

Machine Learning Algorithms in Predicting Hospital Readmissions in Sickle Cell Disease

Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study

Academic, Phase I/II Trial on T Cells Expressing a Second Generation, CD19-Specific Chimeric Antigen Receptor (CAR) and Inducible Caspase 9 Safety Switch for the Treatment of B-Cell Precursor Acute Lymphoblastic Leukemia (BCP-ALL) and B-Cell Non-Hodgkin Lymphoma (B-NHL) in Children

Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene

Expression Profiling of mRNA By Next Generation Sequencing and the Development of Algorithm for Predicting Response in Acute Myeloid Leukemia

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

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