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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

Germline Mutations Predispose to Familial Myeloproliferative Neoplasms

Stephanie Berg, DO, Manuel O. Diaz, MD, Sucha Nand, MD, Jiwang Zhang, MD

Blood (2019) 134 (Supplement_1): 2969.

https://doi.org/10.1182/blood-2019-124805

Published: 2019

Journal Articles

Genome-Wide Methylation Analysis Using the Digital Restriction Enzyme Analysis of Methylation for Stratification of Patients with Juvenile Myelomonocytic Leukemia

Hironobu Kitazawa, Hideki Muramatsu, MD PhD, Norihiro Murakami, MD PhD, Yusuke Okuno, MD PhD, Manabu Wakamatsu, MD, Taro Yoshida, MD, Masayuki Imaya, MD, Ayako Yamamori, MD, Shunsuke Miwata, MD, Kotaro Narita, MD, Motoharu Hamada, MD PhD, Daisuke Ichikawa, MD, Rieko Taniguchi, MD, Nozomu Kawashima, MD PhD, Eri Nishikawa, MD PhD, Atsushi Narita, Nobuhiro Nishio, MD PhD, Seiji Kojima, MD PhD, Yoshiyuki Takahashi, MD PhD

Blood (2019) 134 (Supplement_1): 2973.

https://doi.org/10.1182/blood-2019-127792

Published: 2019

Journal Articles

One Year Data from a Phase 2b Trial of AMT-061 (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B

Steven Pipe, MD, Adam Giermasz, MD PhD, Giancarlo Castaman, MD, Nigel S. Key, MBChB, FRCP, Susan U Lattimore, Frank Leebeek, MD PhD, Wolfgang Miesbach, MD PhD, Michael Recht, MD PhD, Esteban Gomez, MD, Alison Long, Robert Gut, Annette von Drygalski, MD PharmD

Blood (2019) 134 (Supplement_1): 3348.

https://doi.org/10.1182/blood-2019-128765

Published: 2019

Journal Articles

Carfilzomib Induces Cardiotoxicity Via ß5/ß2-Specific Proteasome Subunit Inhibition Pattern

Max A Mendez-Lopez, MD, Andrej Besse, PhD, Bogdan Florea, PhD, Christian Zuppinger, PhD, Herman Overkleeft, PhD, Christoph Driessen, MD, Lenka Besse, PhD

Blood (2019) 134 (Supplement_1): 3110.

https://doi.org/10.1182/blood-2019-127987

Published: 2019

Journal Articles

Suicide Gene Therapy with a CD33 Targeted AAV6 Vector Expressing an Inducible Caspase-9 Suicide Gene Is Therapeutic in a Xenotransplantation Model of Acute Myeloid Leukemia

Nusrat Khan, MSc, Sridhar Bammidi, Jayandharan Giridhara Rao, PhD

Blood (2019) 134 (Supplement_1): 3351.

https://doi.org/10.1182/blood-2019-125222

Published: 2019

Journal Articles

Seroprevalence of Pre-Existing Nabs Against AAV1, 2, 5, 6 and 8 in South African Hemophilia B Patient Population

Anna Majowicz, PhD, Nolukholo Ncete, MBBCH, Floris van Waes, MSc, Nikki Timmer, BSc, Sander J van Deventer, MD PhD, Johnny N Mahlangu, MBBCH, MMed, FCPath, Valerie Ferreira, PhD

Blood (2019) 134 (Supplement_1): 3353.

https://doi.org/10.1182/blood-2019-128217

Published: 2019

Journal Articles

Examining the Role of CD30 in an Anaplastic Large Cell Lymphoma Mouse Model

Teresa Poggio, Linda Graessel, Stefanie Kreutmair, Cornelius Miething, Dietmar Pfeifer, Geoffroy Andrieux, Melanie Boerries, Marie Follo, Suzanne Turner, Robert Zeiser, MD, Justus Duyster, MD, Anna Lena Illert

Blood (2019) 134 (Supplement_1): 2542.

https://doi.org/10.1182/blood-2019-125589

Published: 2019

Journal Articles

A CRISPR/Cas9 Library Screen in Patients' Leukemia Cells In Vivo

Ehsan Bahrami, PhD, Martin Becker, PhD, Anna-Katharina Wirth, MSc, Jan Phillip Schmid, MSc, Tobias Herold, MD, Rupert Öllinger, PhD, Roland Rad, Irmela Jeremias, MD PhD

Blood (2019) 134 (Supplement_1): 3945.

https://doi.org/10.1182/blood-2019-124288

Published: 2019

Journal Articles

Transcriptional Regulation of Hematopoietic Stem Cells in Aging and Myelodysplastic Syndrome Reveals DDIT3 As a Potential Driver of Transformation

Blood (2019) 134 (Supplement_1): 3764.

https://doi.org/10.1182/blood-2019-122686

Published: 2019

Journal Articles

Diagnostic 2-Gene Classifier in Early-Stage Mycosis Fungoides: A Retrospective Multicenter Study

Pia Rude Nielsen, MD, Jens Ole Eriksen, MSc, PhD, Lise M. Lindahl, MD PhD, Ulrike Wehkamp, MD, Gitte Rinds Andersen, MSc, PhD, Michael Bzorek, Kirsten Grønbæk, Professor, MD DMSc, Niels Odum, MD DMSci, Thomas Litman, Prof., Lise Mette Rahbek Gjerdrum, MD PhD

Blood (2019) 134 (Supplement_1): 2772.

https://doi.org/10.1182/blood-2019-122031

Published: 2019

Journal Articles

Loss of ZBTB7A Enhances Glycolysis and Beta Oxidation in Myeloid Leukemia

Enric Redondo Monte, MSc, BSc, Anja Wilding, MSc, Georg Leubolt, MSc, Paul Kerbs, MSc, Johannes Bagnoli, Wolfgang Hiddemann, MD PhD, Wolfgang Enard, PhD, Sebastian Theurich, MD, Philipp A. Greif, MD PD

Blood (2019) 134 (Supplement_1): 1453.

https://doi.org/10.1182/blood-2019-128378

Published: 2019

Journal Articles

Identification of T-Cell Receptors Specific to Antigens Presented By HLA-B4002 and B5401 in Acquired Aplastic Anemia

Kohei Hosokawa, MD PhD, Eiji Kobayashi, PhD, Yoshiki Akatsuka, MD PhD, Luis Espinoza, MD PhD, Noriharu Nakagawa, MD, Tanabe Mikoto, MD, Noriaki Tsuji, MD, Takeshi Yoroidaka, MD, Hiroki Mizumaki, MD, Thi Mai Anh Nguyen, MD, Takamasa Katagiri, PhD, Kiyomi Shitaoka, PhD, Hiroshi Hamana, PhD, Hiroyuki Kishi, PhD, Shinji Nakao, MD PhD

Blood (2019) 134 (Supplement_1): 3751.

https://doi.org/10.1182/blood-2019-125685

Published: 2019

Journal Articles

NUP98-HOXA9 -Initiates Molecular and Biologic Features of Disease Progression in a Humanized Model of CML

Ivan Sloma, PharmD, PhD, Philip Beer, PhD FRCPath, MRCP, Christophe Desterke, PhD, Elizabeth Bulaeva, Misha Bilenky, Michelle Moksa, Kamini Raghuram, Cedric Brimacombe, Karen Lambie, Ali G Turhan, MD PhD, Orianne Wagner-Ballon, MD PhD, Philippe Gaulard, MD PhD, R. Keith Humphries, MD PhD, Martin Hirst, PhD BS, Connie J. Eaves, PhD

Blood (2019) 134 (Supplement_1): 4139.

https://doi.org/10.1182/blood-2019-127839

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

Germline Mutations Predispose to Familial Myeloproliferative Neoplasms

Genome-Wide Methylation Analysis Using the Digital Restriction Enzyme Analysis of Methylation for Stratification of Patients with Juvenile Myelomonocytic Leukemia

One Year Data from a Phase 2b Trial of AMT-061 (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B

Carfilzomib Induces Cardiotoxicity Via ß5/ß2-Specific Proteasome Subunit Inhibition Pattern

Suicide Gene Therapy with a CD33 Targeted AAV6 Vector Expressing an Inducible Caspase-9 Suicide Gene Is Therapeutic in a Xenotransplantation Model of Acute Myeloid Leukemia

Seroprevalence of Pre-Existing Nabs Against AAV1, 2, 5, 6 and 8 in South African Hemophilia B Patient Population

Examining the Role of CD30 in an Anaplastic Large Cell Lymphoma Mouse Model

A CRISPR/Cas9 Library Screen in Patients' Leukemia Cells In Vivo

Transcriptional Regulation of Hematopoietic Stem Cells in Aging and Myelodysplastic Syndrome Reveals DDIT3 As a Potential Driver of Transformation

Diagnostic 2-Gene Classifier in Early-Stage Mycosis Fungoides: A Retrospective Multicenter Study

Loss of ZBTB7A Enhances Glycolysis and Beta Oxidation in Myeloid Leukemia

Identification of T-Cell Receptors Specific to Antigens Presented By HLA-B4002 and B5401 in Acquired Aplastic Anemia

NUP98-HOXA9 -Initiates Molecular and Biologic Features of Disease Progression in a Humanized Model of CML

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American Society of Hematology

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ASH Publications

American Society of Hematology

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