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Journal Articles

Translating an HPFH-Mechanism into Oral Small Molecule Therapy for Beta-Hemoglobinopathies: Clinical Proof-of-Principle

Robert Molokie, Santosh L. Saraf, Lani Krauz, Michael Pacini, David Chan, Alana Aziz-Bradley, Rebeca Gansari, Taif Hassan, Philip Woost, James Jacobberger, Victor R. Gordeuk, Donald E. Lavelle, Joseph DeSimone, Yogenthiran Saunthararajah

Blood (2023) 142 (Supplement 1): 2524.

https://doi.org/10.1182/blood-2023-186228

Published: 2023

Journal Articles

Protein Phosphorylation Mechanisms Underlying Chronic Pain in Sickle Cell Disease

Ying He, PhD, Diana J. Wilkie, PhD RN, FAAN, Robert E. Molokie, MD, Joseph DeSimone, PhD, Zaijie J. Wang, PhD

Blood (2018) 132 (Supplement 1): 3646.

https://doi.org/10.1182/blood-2018-99-115263

Published: 2018

Journal Articles

The LSD1 Inhibitor RN-1 Increases γ-Globin Expression in Baboons By Targeting an Early Event Responsible for γ-Globin Repression

Kestis Vaitkus, Vinzon Ibanez, B.S., Maria Armila Ruiz, B.S., Ramasamy Jagadeeswaran, PhD, Yogenthiran Saunthararajah, MD, James Douglas Engel, PhD, Joseph DeSimone, PhD, Angela Rivers, MD, Donald Lavelle, PhD

Blood (2018) 132 (Supplement 1): 1054.

https://doi.org/10.1182/blood-2018-99-113539

Published: 2018

Journal Articles

Efficacy and safety of long-term RN-1 treatment to increase HbF in baboons

Vinzon Ibanez, Kestis Vaitkus, Angela Rivers, Robert Molokie, Shuaiying Cui, James Douglas Engel, Joseph DeSimone, Donald Lavelle

Blood (2017) 129 (2): 260–263.

https://doi.org/10.1182/blood-2016-10-746727

Published: 2017

Journal Articles

Phase 1 Evaluation of Oral Tetrahydrouridine-Decitabine As Non-Cytotoxic Epigenetic Disease Modification for Sickle Cell Disease

Robert E Molokie, MD, Donald Lavelle, PhD, Michel Gowhari, DO, Michael Pacini, PharmD, Lani Krauz, Johara Hassan, MD, Vinzon Ibanez, Maria A Ruiz, Daisy Pacelli, Sherry Fada, Mitch A. Phelps, PhD, Santosh L. Saraf, MD, Lewis L. Hsu, MD PhD, Victor R. Gordeuk, MD, Joseph DeSimone, PhD, Yogenthiran Saunthararajah, MD

Blood (2016) 128 (22): 124.

https://doi.org/10.1182/blood.V128.22.124.124

Published: 2016

Journal Articles

Efficacy and Safety of Long-Term Treatment with the LSD1 Inhibitor RN-1 to Increase HbF in Normal, Non-Anemic Baboons

Kestis Vaitkus, Vinzon Ibanez, Robert E Molokie, MD, Angela Rivers, MD PhD, Joseph DeSimone, PhD, Donald Lavelle, PhD

Blood (2016) 128 (22): 324.

https://doi.org/10.1182/blood.V128.22.324.324

Published: 2016

Journal Articles

Mitophagy Induction Is a Potential Therapeutic Approach for Sickle Cell Disease

Ramasamy Jagadeeswaran, PhD, Benjamin Alejandro Vazquez, BS, Vinzon Ibanez, Maria A Ruiz, Robert E Molokie, MD, Joseph DeSimone, PhD, Donald Lavelle, PhD, Angela Rivers, MD PhD

Blood (2016) 128 (22): 267.

https://doi.org/10.1182/blood.V128.22.267.267

Published: 2016

Journal Articles

Investigation of Two Combination HbF Induction Regimens, RN-1 and Hydroxyurea Versus RN-1 and Decitabine, in a Humanized Sickle Cell Mouse Model

Ramasamy Jagadeeswaran, PhD, Yash Agarwal, Vinzon Ibanez, Maria Armila Ruiz, Kestis Vaitkus, Joseph DeSimone, PhD, Donald Lavelle, PhD, Angela Rivers, MD PhD

Blood (2015) 126 (23): 3386.

https://doi.org/10.1182/blood.V126.23.3386.3386

Published: 2015

Journal Articles

The LSD1 Inhibitor RN-1 Recapitulates the Fetal Pattern of Hemoglobin Synthesis in Anemic and Normal, Non-Anemic Baboons

Angela Rivers, MD PhD, Kestis Vaitkus, Vinzon Ibanez, Maria Armila Ruiz, Ramaswamy Jagadeeswaran, PhD, Joseph DeSimone, PhD, Donald Lavelle, PhD

Blood (2015) 126 (23): 642.

https://doi.org/10.1182/blood.V126.23.642.642

Published: 2015

Journal Articles

The LSD1 inhibitor RN-1 induces fetal hemoglobin synthesis and reduces disease pathology in sickle cell mice

Shuaiying Cui, Kim-Chew Lim, Lihong Shi, Mary Lee, Natee Jearawiriyapaisarn, Greggory Myers, Andrew Campbell, David Harro, Shigeki Iwase, Raymond C. Trievel, Angela Rivers, Joseph DeSimone, Donald Lavelle, Yogen Saunthararajah, James Douglas Engel

Blood (2015) 126 (3): 386–396.

https://doi.org/10.1182/blood-2015-02-626259

Published: 2015

Includes: Supplemental data

Journal Articles

Combination with Thu to Address Pharmacologic Limitations of Decitabine, Interim PK/PD from a Phase 1/2 Clinical Trial of Oral Thu-Decitabine in Sickle Cell Disease

Donald Lavelle, PhD, Michel Gowhari, DO, Michael Pacini, PharmD, Lani Krauz, RN, Johara Hassan, MD, Vinzon Ibanez, Maria A. Ruiz, MSc, Daisy Pacelli, BSc, Chaher Alhandalous, MD, Mitch A. Phelps, PhD, Kathleen Fenner, BS,MS, Reda Z. Mahfouz, MDPhD, John Barnard, Lewis L. Hsu, MD PhD, Victor R. Gordeuk, MD, Joseph DeSimone, PhD, Yogen Saunthararajah, MD, Robert E. Molokie, MD

Blood (2014) 124 (21): 90.

https://doi.org/10.1182/blood.V124.21.90.90

Published: 2014

Journal Articles

The Tet Dioxygenase Co-Factor Ascorbic Acid Reduces DNA Methylation and Increases Expression of the γ-Globin Gene and Acts in a Combinatorial Manner with HbF-Inducing Drugs Targeting Repressive Epigenetic Modifications

Maria Armila Ruiz, B.S., Angela Rivers, MD PhD, Kestis Vaitkus, Vinzon Ibanez, Robert E. Molokie, MD, Joseph DeSimone, PhD, Donald Lavelle, PhD

Blood (2014) 124 (21): 334.

https://doi.org/10.1182/blood.V124.21.334.334

Published: 2014

Journal Articles

RN-1, a Potent and Selective LSD1 Inhibitor, Induces High Levels of Fetal Hemoglobin (HbF) in Anemic Baboons ( P. anubis )

Angela Rivers, MD PhD, Kestis Vaitkus, Maria Armila Ruiz, B.S., Vinzon Ibanez, Tatiana Kouznetsova, M.S., Joseph DeSimone, PhD, Donald Lavelle, PhD

Blood (2014) 124 (21): 336.

https://doi.org/10.1182/blood.V124.21.336.336

Published: 2014

Journal Articles

Elevated Levels Of γ-Globin Gene Promoter 5-Hydroxymethylcytosine are Associated With Increased DNA Hypomethylation and HbF Expression

Maria Armila Ruiz, BS, Angela Rivers, MD, PhD, Kestis Vaitkus, Tatiana Kouznetsova, M.S., Nadim Mahmud, MD, PhD, Joseph DeSimone, PhD, Donald Lavelle, PH.D.

Blood (2013) 122 (21): 438.

https://doi.org/10.1182/blood.V122.21.438.438

Published: 2013

Journal Articles

RN-1, a New and Potent Lsd-1 Inhibitor, Increases γ-Globin Expression In a Humanized Sickle Cell Mouse Model

Angela Rivers, MD, PhD, Maria Armila Ruiz, BS, Kestis Vaitkus, Tatiana Kouznetsova, M.S., Joseph DeSimone, PhD, Donald Lavelle, Ph.D.

Blood (2013) 122 (21): 561.

https://doi.org/10.1182/blood.V122.21.561.561

Published: 2013

Journal Articles

Pain Medication: Time to First Dose in Sickle Cell Acute Care in Two Settings of a Large Urban Hospital

Bonnye Johnson, MS, RN, Dawn Lei, BS, Nicholas F Furtado, MD, Saman K. Hashmi, MD, Robert Molokie, MD, Joseph DeSimone, PhD, Johara Hassan, MD, Michel Gowhari, DO, Tara Gordon, RN, Darcel Dowell, RN, Glenda Pendarvis, RN, Heather Prendergast, MD, Victor R. Gordeuk, MD, Lewis L. Hsu, MD, PhD

Blood (2012) 120 (21): 4693.

https://doi.org/10.1182/blood.V120.21.4693.4693

Published: 2012

Journal Articles

Dynamic Regulation of 5-Hydroxymethylcytosine At the γ-Globin Promoter During Erythroid Differentiation

Maria Armila Ruiz, B.S., Kestis Vaitkus, Aparna Vasanthakumar, PhD, Angela Rivers, MD, PhD, Tatiana Kouznetsova, M.S., Joseph DeSimone, PhD, Lucy A. Godley, MD, PhD, Donald Lavelle, Ph.D.

Blood (2012) 120 (21): 824.

https://doi.org/10.1182/blood.V120.21.824.824

Published: 2012

Journal Articles

Combinatorial Effect of Pharmacological Inhibitors of Lsd-1 and DNMT on γ-Globin Synthesis in Cultured Baboon Bone Marrow Erythroid Progenitors

Donald Lavelle, Ph.D., Kestis Vaitkus, Maria Armila Ruiz, B.S., Angela Rivers, MD, PhD, James D. Engel, PhD, Joseph DeSimone, PhD

Blood (2012) 120 (21): 249.

https://doi.org/10.1182/blood.V120.21.249.249

Published: 2012

Journal Articles

Effects of tetrahydrouridine on pharmacokinetics and pharmacodynamics of oral decitabine

Donald Lavelle, Kestis Vaitkus, Yonghua Ling, Maria A. Ruiz, Reda Mahfouz, Kwok Peng Ng, Soledad Negrotto, Nicola Smith, Pramod Terse, Kory J. Engelke, Joseph Covey, Kenneth K. Chan, Joseph DeSimone, Yogen Saunthararajah

Blood (2012) 119 (5): 1240–1247.

https://doi.org/10.1182/blood-2011-08-371690

Published: 2012

Includes: Supplemental data

Journal Articles

Quantitative Analysis of Tissue and Developmental Stage-Specific Differences in DNA Methylation throughout the β-Globin Gene Locus by DNA MassARRAY,

Maria Armila Ruiz, B.S., Kestutis Vaitkus, B.S., Tatiana Kouznetsova, B.S., Joseph DeSimone, PhD, Yogen Saunthararajah, MD, Donald Lavelle, Ph.D.

Blood (2011) 118 (21): 3196.

https://doi.org/10.1182/blood.V118.21.3196.3196

Published: 2011

NARROW

Translating an HPFH-Mechanism into Oral Small Molecule Therapy for Beta-Hemoglobinopathies: Clinical Proof-of-Principle

Protein Phosphorylation Mechanisms Underlying Chronic Pain in Sickle Cell Disease

The LSD1 Inhibitor RN-1 Increases γ-Globin Expression in Baboons By Targeting an Early Event Responsible for γ-Globin Repression

Efficacy and safety of long-term RN-1 treatment to increase HbF in baboons

Phase 1 Evaluation of Oral Tetrahydrouridine-Decitabine As Non-Cytotoxic Epigenetic Disease Modification for Sickle Cell Disease

Efficacy and Safety of Long-Term Treatment with the LSD1 Inhibitor RN-1 to Increase HbF in Normal, Non-Anemic Baboons

Mitophagy Induction Is a Potential Therapeutic Approach for Sickle Cell Disease

Investigation of Two Combination HbF Induction Regimens, RN-1 and Hydroxyurea Versus RN-1 and Decitabine, in a Humanized Sickle Cell Mouse Model

The LSD1 Inhibitor RN-1 Recapitulates the Fetal Pattern of Hemoglobin Synthesis in Anemic and Normal, Non-Anemic Baboons

The LSD1 inhibitor RN-1 induces fetal hemoglobin synthesis and reduces disease pathology in sickle cell mice

Combination with Thu to Address Pharmacologic Limitations of Decitabine, Interim PK/PD from a Phase 1/2 Clinical Trial of Oral Thu-Decitabine in Sickle Cell Disease

The Tet Dioxygenase Co-Factor Ascorbic Acid Reduces DNA Methylation and Increases Expression of the γ-Globin Gene and Acts in a Combinatorial Manner with HbF-Inducing Drugs Targeting Repressive Epigenetic Modifications

RN-1, a Potent and Selective LSD1 Inhibitor, Induces High Levels of Fetal Hemoglobin (HbF) in Anemic Baboons ( P. anubis )

Elevated Levels Of γ-Globin Gene Promoter 5-Hydroxymethylcytosine are Associated With Increased DNA Hypomethylation and HbF Expression

RN-1, a New and Potent Lsd-1 Inhibitor, Increases γ-Globin Expression In a Humanized Sickle Cell Mouse Model

Pain Medication: Time to First Dose in Sickle Cell Acute Care in Two Settings of a Large Urban Hospital

Dynamic Regulation of 5-Hydroxymethylcytosine At the γ-Globin Promoter During Erythroid Differentiation

Combinatorial Effect of Pharmacological Inhibitors of Lsd-1 and DNMT on γ-Globin Synthesis in Cultured Baboon Bone Marrow Erythroid Progenitors

Effects of tetrahydrouridine on pharmacokinetics and pharmacodynamics of oral decitabine

Quantitative Analysis of Tissue and Developmental Stage-Specific Differences in DNA Methylation throughout the β-Globin Gene Locus by DNA MassARRAY,

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American Society of Hematology

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American Society of Hematology

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