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Optimization of the capsid of recombinant adeno-associated virus 2 (AAV2) vectors: the final threshold?

PloS one, 2013-03, Vol.8 (3), p.e59142

2013

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A new target for fetal hemoglobin reactivation

Haematologica (Roma), 2019-12, Vol.104 (12), p.2325-2327

2019

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Rationally designed capsid and transgene cassette of AAV6 vectors for dendritic cell‐based cancer immunotherapy

Immunology and cell biology, 2014-02, Vol.92 (2), p.116-123

2014

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

Blood, 2015-07, Vol.126 (3), p.386-396

2015

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Combinatorial targeting of epigenome-modifying enzymes with decitabine and RN-1 synergistically increases HbF

Blood advances, 2023-08, Vol.7 (15), p.3891-3902

2023

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Drug-loaded sickle cells programmed ex vivo for delayed hemolysis target hypoxic tumor microvessels and augment tumor drug delivery

Journal of controlled release, 2013-10, Vol.171 (2), p.184-192

2013

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Cognition affected in young children with serum ferritin <17 μg/L

The Journal of pediatrics, 2020-05, Vol.220, p.264-267

Rivers, Angela

2020

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

Blood, 2017-01, Vol.129 (2), p.260-263

2017

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The LSD1 inhibitor RN-1 recapitulates the fetal pattern of hemoglobin synthesis in baboons (P. anubis)

Haematologica (Roma), 2016-06, Vol.101 (6), p.688-697

2016

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Optimizing the transduction efficiency of capsid-modified AAV6 serotype vectors in primary human hematopoietic stem cells in vitro and in a xenograft mouse model in vivo

Cytotherapy (Oxford, England), 2013-08, Vol.15 (8), p.986-998

2013

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Adverse Reactions to Pneumococcal Vaccine in Pediatric and Adolescent Patients with Sickle Cell Disease

Pharmacotherapy, 2015-07, Vol.35 (7), p.696-700

2015

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High-efficiency transduction of human monocyte-derived dendritic cells by capsid-modified recombinant AAV2 vectors

Vaccine, 2012-06, Vol.30 (26), p.3908-3917

2012

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Evolving treatment paradigms in sickle cell disease

Hematology, 2017-12, Vol.2017 (1), p.440-446

2017

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Optimized adeno-associated virus (AAV)-protein phosphatase-5 helper viruses for efficient liver transduction by single-stranded AAV vectors: therapeutic expression of factor IX at reduced vector doses

Human gene therapy, 2010-03, Vol.21 (3), p.271-283

2010

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Potential role of LSD1 inhibitors in the treatment of sickle cell disease: a review of preclinical animal model data

American journal of physiology. Regulatory, integrative and comparative physiology, 2018-10, Vol.315 (4), p.R840-R847

2018

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Congenital Cytopenias and Bone Marrow Failure Syndromes

Seminars in perinatology, 2009-02, Vol.33 (1), p.20-28

2009

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T‐cell large granular lymphocytic leukemia successfully treated with oral cyclosporine in a child: Case report and review of literature

Pediatric blood & cancer, 2020-12, Vol.67 (12), p.e28487-n/a

2020

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Nicotinamide Improves Anemia and Decreases Reactive Oxygen Species in Sickle Cell Disease Mice

Blood, 2021-11, Vol.138 (Supplement 1), p.2019-2019

2021

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Reticulocytosis from Stress Erythropoiesis Is a Major Source of Erythrocyte Mitochondrial Retention, Oxygen Consumption and Reactive Oxygen Species in a SCD Mouse Model

Blood, 2022-11, Vol.140 (Supplement 1), p.8239-8240

2022

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A Prospective Study of Genital Herpes Simplex Virus Type 2 Infection in Human Immunodeficiency Virus Type 1 (HIV-1)–Seropositive Women: Correlations with CD4 Cell Count and Plasma HIV-1 RNA Level

Clinical infectious diseases, 2003-01, Vol.36 (2), p.207-211

2003

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Hereditary Spherocytosis with Mitochondrial Retention, Increased Oxidative Stress, and Alterations to Bioactive Membrane Lipids

Journal of pediatric hematology/oncology, 2024-08, Vol.46 (6), p.e457-e462

2024

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Cathepsin B, a Negative Regulator of Autophagy, Identified As a Novel Therapeutic Drug Target in Sickle Cell Disease

Blood, 2020-11, Vol.136 (Supplement 1), p.32-32

2020

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Reticulocyte mitochondrial retention increases reactive oxygen species and oxygen consumption in mouse models of sickle cell disease and phlebotomy-induced anemia

Experimental hematology, 2023-06, Vol.122, p.55-62

2023

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Optimization of recombinant adeno-associated viral vectors for human beta-globin gene transfer and transgene expression

Human gene therapy, 2008-04, Vol.19 (4), p.365-375

2008

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RN-1, a potent and selective lysine-specific demethylase 1 inhibitor, increases γ-globin expression, F reticulocytes, and F cells in a sickle cell disease mouse model

Experimental hematology, 2015-07, Vol.43 (7), p.546-553.e3

2015

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Optimization of the capsid of recombinant adeno-associated virus 2 (AAV2) vectors: the final threshold?

A new target for fetal hemoglobin reactivation

Rationally designed capsid and transgene cassette of AAV6 vectors for dendritic cell‐based cancer immunotherapy

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

Combinatorial targeting of epigenome-modifying enzymes with decitabine and RN-1 synergistically increases HbF

Drug-loaded sickle cells programmed ex vivo for delayed hemolysis target hypoxic tumor microvessels and augment tumor drug delivery

Cognition affected in young children with serum ferritin <17 μg/L

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

The LSD1 inhibitor RN-1 recapitulates the fetal pattern of hemoglobin synthesis in baboons (P. anubis)

Optimizing the transduction efficiency of capsid-modified AAV6 serotype vectors in primary human hematopoietic stem cells in vitro and in a xenograft mouse model in vivo

Adverse Reactions to Pneumococcal Vaccine in Pediatric and Adolescent Patients with Sickle Cell Disease

High-efficiency transduction of human monocyte-derived dendritic cells by capsid-modified recombinant AAV2 vectors

Evolving treatment paradigms in sickle cell disease

Optimized adeno-associated virus (AAV)-protein phosphatase-5 helper viruses for efficient liver transduction by single-stranded AAV vectors: therapeutic expression of factor IX at reduced vector doses

Potential role of LSD1 inhibitors in the treatment of sickle cell disease: a review of preclinical animal model data

Congenital Cytopenias and Bone Marrow Failure Syndromes

T‐cell large granular lymphocytic leukemia successfully treated with oral cyclosporine in a child: Case report and review of literature

Nicotinamide Improves Anemia and Decreases Reactive Oxygen Species in Sickle Cell Disease Mice

Reticulocytosis from Stress Erythropoiesis Is a Major Source of Erythrocyte Mitochondrial Retention, Oxygen Consumption and Reactive Oxygen Species in a SCD Mouse Model

A Prospective Study of Genital Herpes Simplex Virus Type 2 Infection in Human Immunodeficiency Virus Type 1 (HIV-1)–Seropositive Women: Correlations with CD4 Cell Count and Plasma HIV-1 RNA Level

Hereditary Spherocytosis with Mitochondrial Retention, Increased Oxidative Stress, and Alterations to Bioactive Membrane Lipids

Cathepsin B, a Negative Regulator of Autophagy, Identified As a Novel Therapeutic Drug Target in Sickle Cell Disease

Reticulocyte mitochondrial retention increases reactive oxygen species and oxygen consumption in mouse models of sickle cell disease and phlebotomy-induced anemia

Optimization of recombinant adeno-associated viral vectors for human beta-globin gene transfer and transgene expression

RN-1, a potent and selective lysine-specific demethylase 1 inhibitor, increases γ-globin expression, F reticulocytes, and F cells in a sickle cell disease mouse model

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