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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

Single Nucleotide Polymorphisms in SAR1A Codon Regions Associated with Hydroxyurea Response in Sickle Cell Disease and Potentially Influenced in Mirnas Binding

Chutima Kumkhaek, PhD, Christine Kim, James G. Taylor, MD, Jianqiong Zhu, MD, Wulin Aerbajinai, MD, Griffin Rodgers, MD

Blood (2019) 134 (Supplement_1): 987.

https://doi.org/10.1182/blood-2019-124965

Published: 2019

Journal Articles

Single-Cell Analysis Based Dissection of Clonality in Myelofibrosis

Blood (2019) 134 (Supplement_1): 469.

https://doi.org/10.1182/blood-2019-123367

Published: 2019

Journal Articles

Ultra-Accurate Assessment of Pretreatment ABL1 Kinase Domain (KD) Mutations in Patients (pts) with Newly Diagnosed Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ ALL) Using Duplex Sequencing (DS)

Blood (2019) 134 (Supplement_1): 2578.

https://doi.org/10.1182/blood-2019-125131

Published: 2019

Journal Articles

The Broad Spectrum of TP53 Variants in CLL: NGS Analysis of 573 Pathogenic TP53 Variants

Blood (2019) 134 (Supplement_1): 3021.

https://doi.org/10.1182/blood-2019-130907

Published: 2019

Journal Articles

Impact of the Association between Tgl and EZH2 Mutations (Y641N/Y641F/Y641H/Y641S) on Disease Free Survival of Patients with Diffuse Large B-Cell Lymphoma

Myrna Candelaria, MD PharmD, Adriana Palacios Campos, MD, Olga Gutierrez Hernandez, QFB, Alejandro Aviles, MD, Uvi Cancino-Ramos, MD, Lucia Taja Chayeb, PhD, Osvaldo García-Perez, MD, David Cantú de León

Blood (2019) 134 (Supplement_1): 5222.

https://doi.org/10.1182/blood-2019-125906

Published: 2019

Journal Articles

Changes in N-Glycosylation Induced By Somatic Hypermutation Modulate the Antigen Reactivity of the Immunoglobulin Receptors in CLL Stereotyped Subset #201

Maria Gounari, Anastasia Iatrou, Kostantia Kotta, Ioannis Sarrigeorgiou, Peggy Lymberi, Anastasia Chatzidimitriou, Kostas Stamatopoulos

Blood (2019) 134 (Supplement_1): 1733.

https://doi.org/10.1182/blood-2019-127852

Published: 2019

Journal Articles

Transplanted Vasoactive Intestinal Peptide-Secreting Donor Plasmacytoid Dendritic Cells Migrate to Lymphoid Organs and GvHD Target Organs

Jingru Zhu, Yitong Wang, MD, Jingxia Li, MD, Yiwen Li, Alina Ulezko Antonova, Cynthia R. Giver, PhD, Jianming Li, PhD, Shuhua Wang, MD, Edmund K. Waller, PhDMD

Blood (2019) 134 (Supplement_1): 1918.

https://doi.org/10.1182/blood-2019-129628

Published: 2019

Journal Articles

Next Generation Sequencing May be Helpful in Designing Novel Combinations Among Heavily Pretreated Myeloma Patients, Refractory to All Available Approved Anti-Myeloma Drugs

Guldane Cengiz Seval, Isinsu Kuzu, Seher Yuksel, Gulsah Kaygusuz, Klara Dalva, MD, Muhit Ozcan, Meral Beksac, MD

Blood (2019) 134 (Supplement_1): 3197.

https://doi.org/10.1182/blood-2019-127541

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

The Factor VIII Variant X5 Enhances Hemophilia a Gene Therapy Efficiency By Its Improved Secretion

Franziska Horling, PhD, Johannes Lengler, PhD, Wenjing Cao, PhD, Biao Dong, PhD, Bagirath Gangadharan, Maurus De La Rosa, PhD, Werner Hoellriegl, PhD, Birgit M Reipert, PhD, Friedrich Scheiflinger, Weidong Xiao, PhD, Hanspeter Rottensteiner

Blood (2019) 134 (Supplement_1): 3356.

https://doi.org/10.1182/blood-2019-126489

Published: 2019

Journal Articles

Universal' Fratricide-Resistant CAR T Cells Against T Cell Leukemia Generated By Coupled & Uncoupled Deamination Mediated Base Editing

Christos Georgiadis, PhD, Jane Rasaiyaah, PhD, Soragia Athina Gkazi, PhD, Roland Preece, Annie Etuk, PhD, Abraham Christi, Waseem Qasim, MBBS, PhD

Blood (2019) 134 (Supplement_1): 3219.

https://doi.org/10.1182/blood-2019-130057

Published: 2019

Journal Articles

Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 4 Years Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B

Wolfgang Miesbach, MD PhD, Karina Meijer, Michiel Coppens, MD, Peter Kampmann, MD, Dr. Klamroth, Roger Schutgens, MD PhD MSc, Giancarlo Castaman, MD, Erhard Seifried, MD PhD, Joachim Schwaeble, Halvard Bönig, Eileen K Sawyer, PhD, Frank W.G. Leebeek, MD PhD

Blood (2019) 134 (Supplement_1): 2059.

https://doi.org/10.1182/blood-2019-122535

Published: 2019

Journal Articles

Hemophilia A Dogs Tolerant to Human Factor VIII Provide a Unique Model to Determine Efficacy and Safety of AAV Delivery of Novel Factor VIII Variants

Giang N. Nguyen, BS, Lauren E. Wimsey, DVM, Elizabeth P. Merricks, PhD, Katherine P. Ponder, MD, Timothy C. Nichols, MD, Denise E. Sabatino, PhD

Blood (2019) 134 (Supplement_1): 3628.

https://doi.org/10.1182/blood-2019-126085

Published: 2019

Journal Articles

Gene Editing and mRNA-Based Therapy: Two Complementary Therapeutic Approaches for the Treatment of Patients with Xmen Disease

Julie Brault, PhD PharmD, Ezekiel Bello, Ronald J. Meis, PhD, Taylor Liu, Sherry M. Koontz, BS, Kennichi Dowdell, PhD, Linhong Li, PhD, Cornell Allen, MS, Narda Theobald, BS, Janet Lee, BS, Aaron B. Clark, BS, Juan C. Ravell, MD, Michael Lenardo, MD, Gary A. Dahl, PhD, Harry L. Malech, MD, Suk See De Ravin, MD, PhD

Blood (2019) 134 (Supplement_1): 4637.

https://doi.org/10.1182/blood-2019-129062

Published: 2019

Journal Articles

The Translational Switch of MYC Protein Aliases in Myeloma Tumor Cells

Erming Tian, PhD MBA, Jeffery R. Sawyer, Ricky D. Edmondson, Maurizio Zangari, MD, Christopher P. Wardell, PhD MSc, BSc, Sharmilan Thanendrarajan, MD, Carolina D. Schinke, MD, Frits van Rhee, MD PhD

Blood (2019) 134 (Supplement_1): 4390.

https://doi.org/10.1182/blood-2019-131850

Published: 2019

Journal Articles

Comparison the Distinct Clinical Outcome with TP53 Mutation Status in MDS and AML :an Retrospective Study

Xinrong Xiang, MD, Yang Ou, Yu Wu, MD

Blood (2019) 134 (Supplement_1): 5405.

https://doi.org/10.1182/blood-2019-124622

Published: 2019

Journal Articles

ASXL1 Mutations Detectable at Diagnosis May Predict Response to Imatinib in Patients with Chronic Myeloid Leukemia

Marcin M Machnicki, MSc, Monika Pepek, MSc, Iwona Solarska, PhD, Joanna Niesiobedzka-Krezel, MD PhD, Ilona Seferynska, MD PhD, Joanna Gora Tybor, MD PhD, Magdalena Zawada, PhD, Tomasz Sacha, MD PhD, Waldemar Sawicki, MD PhD, Ewa Wasilewska, MD PhD, Katarzyna Pruszczyk, MD, Malgorzata Rydzanicz, PhD, Rafal Ploski, MD PhD, Tomasz Skorski, MD PhD, Tomasz Stoklosa, MD PhD

Blood (2019) 134 (Supplement_1): 4148.

https://doi.org/10.1182/blood-2019-129834

Published: 2019

Journal Articles

Determining Resistance Mechanisms in BRAF- mutated Multiple Myeloma

Johannes M. Waldschmidt, MD, Jake A. Kloeber, Tushara Vijaykumar, Antonis Kokkalis, PhD MSc, Praveen Anand, PhD, Sayalee Potdar, Julia Frede, PhD MPhil, Monica S. Nair, Valeriya Dimitrova, Nikhil C. Munshi, MD, Kenneth C. Anderson, MD, Andrew J. Yee, MD, Noopur S. Raje, MD, Birgit Knoechel, MD PhD, Jens G. Lohr, MD PhD

Blood (2019) 134 (Supplement_1): 316.

https://doi.org/10.1182/blood-2019-130313

Published: 2019

Journal Articles

Detection of Multiple Recurrent Novel BCL2 Mutations Co-Occurring with BCL2 Gly101Val in Patients with Chronic Lymphocytic Leukemia on Long Term Venetoclax

Piers Blombery, MBBS, Ella Thompson, PhD BSc, Tamia Nguyen, Xiangting Chen, PhD, Michelle McBean, Richard W Birkinshaw, PhD, Peter E Czabotar, PhD, Rachel Thijssen, Mary Ann Anderson, MBBS, PhD FRACP, FRCPA, John F. Seymour, MBBS, PhD FRACP, David Alan Westerman, MBBS, FRCPA, FRACP, David C.S. Huang, MBBS MRCP PhD, Andrew W. Roberts, MBBS, PhD

Blood (2019) 134 (Supplement_1): 171.

https://doi.org/10.1182/blood-2019-125457

Published: 2019

NARROW

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

Single Nucleotide Polymorphisms in SAR1A Codon Regions Associated with Hydroxyurea Response in Sickle Cell Disease and Potentially Influenced in Mirnas Binding

Single-Cell Analysis Based Dissection of Clonality in Myelofibrosis

Ultra-Accurate Assessment of Pretreatment ABL1 Kinase Domain (KD) Mutations in Patients (pts) with Newly Diagnosed Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ ALL) Using Duplex Sequencing (DS)

The Broad Spectrum of TP53 Variants in CLL: NGS Analysis of 573 Pathogenic TP53 Variants

Impact of the Association between Tgl and EZH2 Mutations (Y641N/Y641F/Y641H/Y641S) on Disease Free Survival of Patients with Diffuse Large B-Cell Lymphoma

Changes in N-Glycosylation Induced By Somatic Hypermutation Modulate the Antigen Reactivity of the Immunoglobulin Receptors in CLL Stereotyped Subset #201

Transplanted Vasoactive Intestinal Peptide-Secreting Donor Plasmacytoid Dendritic Cells Migrate to Lymphoid Organs and GvHD Target Organs

Next Generation Sequencing May be Helpful in Designing Novel Combinations Among Heavily Pretreated Myeloma Patients, Refractory to All Available Approved Anti-Myeloma Drugs

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

The Factor VIII Variant X5 Enhances Hemophilia a Gene Therapy Efficiency By Its Improved Secretion

Universal' Fratricide-Resistant CAR T Cells Against T Cell Leukemia Generated By Coupled & Uncoupled Deamination Mediated Base Editing

Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 4 Years Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B

Hemophilia A Dogs Tolerant to Human Factor VIII Provide a Unique Model to Determine Efficacy and Safety of AAV Delivery of Novel Factor VIII Variants

Gene Editing and mRNA-Based Therapy: Two Complementary Therapeutic Approaches for the Treatment of Patients with Xmen Disease

The Translational Switch of MYC Protein Aliases in Myeloma Tumor Cells

Comparison the Distinct Clinical Outcome with TP53 Mutation Status in MDS and AML :an Retrospective Study

ASXL1 Mutations Detectable at Diagnosis May Predict Response to Imatinib in Patients with Chronic Myeloid Leukemia

Determining Resistance Mechanisms in BRAF- mutated Multiple Myeloma

Detection of Multiple Recurrent Novel BCL2 Mutations Co-Occurring with BCL2 Gly101Val in Patients with Chronic Lymphocytic Leukemia on Long Term Venetoclax

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American Society of Hematology

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ASH Publications

American Society of Hematology

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