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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

Core Transcriptional Regulatory Circuitries in AML

Maxim Pimkin, MD PhD, Juliana Xavier Ferrucio, PhD, Neekesh Vijay Dharia, MD PhD, Taku Harada, Andrew Kossenkov, Selma Elsarrag, Jazmin Ewers, Charles Y Lin, PhD, Kimberly Stegmaier, MD, Stuart H. Orkin, MD

Blood (2019) 134 (Supplement_1): 280.

https://doi.org/10.1182/blood-2019-129297

Published: 2019

Journal Articles

Fitusiran, an RNAi Therapeutic Targeting Antithrombin to Restore Hemostatic Balance in Patients with Hemophilia a or B with or without Inhibitors: Management of Acute Bleeding Events

Steven Pipe, MD, Margaret V. Ragni, MD MPH, Claude Négrier, MD PhD, Qifeng Yu, PhD, Naghmana Bajwa, MD, John Caminis, MD, Baisong Mei, MD PhD, Shauna R. Andersson, MD PhD

Blood (2019) 134 (Supplement_1): 1138.

https://doi.org/10.1182/blood-2019-124108

Published: 2019

Journal Articles

Synthetic Lethality-Based Approach Identified EED and BRD4 As Critical Survival Factorsin Acte Myeloid Leukemia with Monosomy 7

Kensuke Matsuda, MD, Yuki Kagoya, MD PhD, Sho Yamazaki, MD PhD, Masashi Miyauchi, MD PhD, Mineo Kurokawa, MD PhD

Blood (2019) 134 (Supplement_1): 1233.

https://doi.org/10.1182/blood-2019-123595

Published: 2019

Journal Articles

Innate Immune Signaling Suppresses Acute Leukemia By Modifying MYC Oncogenic Activity

Tomoya Muto, MD PhD, Maria Guillamot, PhD, Jing Fang, MD PhD, Luna Zea Redondo, MS, Kwangmin Choi, PhD, Yixiao Gong, MSc, Callum S Walker, MSc, Kathleen Hueneman, Lyndsey C Bolanos, BSc, Laura Barreyro, PhD, Lynn Lee, MD, Kenneth D Greis, PhD, Alireza Khodadadi-Jamayran, M.Sc., Amaia Lujambio, PhD, Scott W. Lowe, PhD, Iannis Aifantis, PhD, Daniel T. Starczynowski, PhD

Blood (2019) 134 (Supplement_1): 727.

https://doi.org/10.1182/blood-2019-121423

Published: 2019

Journal Articles

SIRT1 Mediates Enhanced Mitochondrial Oxidative Phosphorylation in Chronic Myelogenous Leukemia Stem Cells

Ajay Abraham, PhD, Shaowei Qiu, MD, Balu K Chacko, PhD, Hui Li, BS, Andrew J Paterson, PhD, Jianbo He, MD, Puneet Agarwal, PhD, Mansi Shah, PhD, Robert S. Welner, PhD, Victor M Darley-Usmar, PhD, Ravi Bhatia, MD

Blood (2018) 132 (Supplement 1): 932.

https://doi.org/10.1182/blood-2018-99-115709

Published: 2018

Journal Articles

IGFBP7 As a Potential Therapeutic Target for AML

Yina Niu, PhD, Shuiyan Wu, MD, Shaoyan Hu, MD PhD

Blood (2018) 132 (Supplement 1): 5134.

https://doi.org/10.1182/blood-2018-99-119528

Published: 2018

Journal Articles

Dependency of Spliceosomal Mutant MDS on Innate Immune Signaling

Stanley C Lee, PhD, Khrystyna North, Chi-Chao Chen, Eunhee Kim, PhD, Sydney X. Lu, MD PhD, Michelle Ki, BSc, Ralph Garippa, PhD, Scott W Lowe, PhD, Robert K. Bradley, PhD, Omar I Abdel-Wahab, MD

Blood (2018) 132 (Supplement 1): 937.

https://doi.org/10.1182/blood-2018-99-111552

Published: 2018

Journal Articles

Transcriptional Control of Memory T Cell Differentiation

Ananda Goldrath, PhD

Blood (2018) 132 (Supplement 1): SCI-7.

https://doi.org/10.1182/blood-2018-99-109536

Published: 2018

Journal Articles

IQGAP1: A Potential Therapeutic Target in Acute Myelogenous Leukemia (AML)

Deepak Sahasrabudhe, MD, John M Ashton, PhD, Andrew G Evans, MD PhD, McMahon Loralee, Balys Merlene, MS, Jane L. Liesveld, MD

Blood (2017) 130 (Supplement 1): 1391.

https://doi.org/10.1182/blood.V130.Suppl_1.1391.1391

Published: 2017

Journal Articles

RNAi and CRISPR/Cas9 Based In Vivo Models for Drug Discovery

Prem Premsrirut, MD PhD, Chia-Lin Wang, PhD, Yu-ting Yang, PhD, Christof Fellmann, PhD, Lukas Dow, PhD, Johannes Zuber, PhD, Scott Lowe, PhD, Marina Pesic, PhD, Katharina Wolter, PhD, Lars Zender, PhD

Blood (2017) 130 (Supplement 1): 5568.

https://doi.org/10.1182/blood.V130.Suppl_1.5568.5568

Published: 2017

Journal Articles

RNAi Screens for Maintenance of Hematopoietic Stem Cell Phenotype During Culture

Jonas Larsson, MD PhD

Blood (2017) 130 (Supplement 1): SCI-45.

https://doi.org/10.1182/blood.V130.Suppl_1.SCI-45.SCI-45

Published: 2017

Journal Articles

Hematopoietic-Specific CSNK2B Loss in Mice Causes Impaired Erythropoiesis

Laura Quotti Tubi, PhD, Sara Canovas Nunes, PhD, Marilena Carrino, PhD, Ketty Gianesin, PhD, Sabrina Manni, PhD, Odile Filhol-Cochet, PhD, Brigitte Boldyreff, PhD, Livio Trentin, MD, Gianpietro Semenzato, MD, Francesco Piazza, MD

Blood (2017) 130 (Supplement 1): 82.

https://doi.org/10.1182/blood.V130.Suppl_1.82.82

Published: 2017

Journal Articles

Perioperative Management in Patients with Hemophilia Receiving Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia

Claude Negrier, MD, Margaret V. Ragni, MD MPH, Pencho Georgiev, MD, Toshko Jelev Lissitchkov, MD PhD, Huy Van Nguyen, PharmD, Kate Madigan, K John Pasi

Blood (2017) 130 (Supplement 1): 2366.

https://doi.org/10.1182/blood.V130.Suppl_1.2366.2366

Published: 2017

Journal Articles

In Vivo Effects of LSD1 Inhibition By Small Chemical Inhibitors in Sickle Cell Mice

Cuong Le, PhD, Greggory Myers, Alawi Habara, Natee Jearawiriyapaisarn, PhD, Mary Lee, PhD, David H.K. Chui, MD, Martin H. Steinberg, MD, James Engel, PhD, Shuaiying Cui, PhD

Blood (2017) 130 (Supplement 1): 968.

https://doi.org/10.1182/blood.V130.Suppl_1.968.968

Published: 2017

Journal Articles

Dot1l Reverses the Abnormal Self-Renewal Conferred on HSPCs through Cohesin Haploinsufficiency By Diminishing Expression of Hoxa7 and Hoxa9

Joseph B Fisher, PhD, Cary Stelloh, Emily Kristine Phillips, BS, Katelyn Heimbruch, BS, Alison E Meyer, PhD, Kirthi Pulakanti, Maureen McNulty, BS, Aaron D Viny, MDMS, Ross L. Levine, MD, John D. Crispino, PhD, Sridhar Rao, MD PhD

Blood (2017) 130 (Supplement 1): 246.

https://doi.org/10.1182/blood.V130.Suppl_1.246.246

Published: 2017

Journal Articles

Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results from a Phase 1 and Phase 1/2 Extension Study in Patients with Inhibitors

K John Pasi, Pencho Georgiev, Tim Mant, Toshko Lissitchkov, Michael Desmond Creagh, David Bevan, Steve Austin, Charles R Hay, Inga Hegemann, Rashid Kazmi, Pratima Chowdary, Margaret V. Ragni, MD, Chang-Heok Soh, Akin Akinc, Angela M. Partisano, Benny Sorenson, Savita Rangarajan

Blood (2016) 128 (22): 1397.

https://doi.org/10.1182/blood.V128.22.1397.1397

Published: 2016

Journal Articles

Argonaute 2 Silencing of Band 3 in Early Reticulocytes

Bloch M Evgenia, Donald R. Branch, PhD

Blood (2016) 128 (22): 1273.

https://doi.org/10.1182/blood.V128.22.1273.1273

Published: 2016

Journal Articles

Effects of RNA Interference on CD70 in Dendritic Cells of Patients with Immune Thrombocytopenia

Zeping Zhou, Sr., MD, Yunlong Wang, Jr., MD, Renchi Yang, MD PhD, Huiyuan Li, PhD

Blood (2016) 128 (22): 1373.

https://doi.org/10.1182/blood.V128.22.1373.1373

Published: 2016

Journal Articles

A Subcutaneously Administered Investigational RNAi Therapeutic (ALN-CC5) Targeting Complement C5 for Treatment of PNH and Complement-Mediated Diseases: Preliminary Phase 1/2 Study Results in Patients with PNH

Anita Hill, Anna Gaya Valls, Morag Griffin, Talha Munir, Anna Borodovsky, Nori Kawahata, Helen Mclean, Kelvin Shi, Angela M. Partisano, Jae Kim, Nader Najafian, Alvaro Urbano

Blood (2016) 128 (22): 3891.

https://doi.org/10.1182/blood.V128.22.3891.3891

Published: 2016

NARROW

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

Core Transcriptional Regulatory Circuitries in AML

Fitusiran, an RNAi Therapeutic Targeting Antithrombin to Restore Hemostatic Balance in Patients with Hemophilia a or B with or without Inhibitors: Management of Acute Bleeding Events

Synthetic Lethality-Based Approach Identified EED and BRD4 As Critical Survival Factorsin Acte Myeloid Leukemia with Monosomy 7

Innate Immune Signaling Suppresses Acute Leukemia By Modifying MYC Oncogenic Activity

SIRT1 Mediates Enhanced Mitochondrial Oxidative Phosphorylation in Chronic Myelogenous Leukemia Stem Cells

IGFBP7 As a Potential Therapeutic Target for AML

Dependency of Spliceosomal Mutant MDS on Innate Immune Signaling

Transcriptional Control of Memory T Cell Differentiation

IQGAP1: A Potential Therapeutic Target in Acute Myelogenous Leukemia (AML)

RNAi and CRISPR/Cas9 Based In Vivo Models for Drug Discovery

RNAi Screens for Maintenance of Hematopoietic Stem Cell Phenotype During Culture

Hematopoietic-Specific CSNK2B Loss in Mice Causes Impaired Erythropoiesis

Perioperative Management in Patients with Hemophilia Receiving Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia

In Vivo Effects of LSD1 Inhibition By Small Chemical Inhibitors in Sickle Cell Mice

Dot1l Reverses the Abnormal Self-Renewal Conferred on HSPCs through Cohesin Haploinsufficiency By Diminishing Expression of Hoxa7 and Hoxa9

Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results from a Phase 1 and Phase 1/2 Extension Study in Patients with Inhibitors

Argonaute 2 Silencing of Band 3 in Early Reticulocytes

Effects of RNA Interference on CD70 in Dendritic Cells of Patients with Immune Thrombocytopenia

A Subcutaneously Administered Investigational RNAi Therapeutic (ALN-CC5) Targeting Complement C5 for Treatment of PNH and Complement-Mediated Diseases: Preliminary Phase 1/2 Study Results in Patients with PNH

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American Society of Hematology

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ASH Publications

American Society of Hematology

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