Area of research
Genetics · Molecular Biology
Research interest
Research interests include Hemoglobinopathies and Related Disorders, Acute Myeloid Leukemia Research, Histone Deacetylase Inhibitors Research, and Virus-based gene therapy research.
OVATION-2: A randomized phase I/II study evaluating the safety and efficacy of IMNN-001 (IL-12 gene therapy) with neo/adjuvant chemotherapy in patients newly-diagnosed with advanced epithelial ovarian cancer
Targeted therapy with nanatinostat and valganciclovir in recurrent EBV-positive lymphoid malignancies: a phase 1b/2 study
Epilepsy: Mitochondrial connections to the ‘Sacred’ disease
Pevonedistat plus azacitidine vs azacitidine alone in higher-risk MDS/chronic myelomonocytic leukemia or low-blast-percentage AML
Treatment and prevention of pathological mitochondrial dysfunction in retinal degeneration and in photoreceptor injury
Randomized phase 2 trial of pevonedistat plus azacitidine versus azacitidine for higher-risk MDS/CMML or low-blast AML
A randomized phase 3 study of ixazomib–dexamethasone versus physician’s choice in relapsed or refractory AL amyloidosis
Pathogenic mitochondrial dysfunction and metabolic abnormalities
Measuring patient-reported physical functioning and fatigue in myelodysplastic syndromes using a modular approach based on EORTC QLQ-C30
Pevonedistat (PEV) + Azacitidine (AZA) Versus AZA Alone As First-Line Treatment for Patients with Higher-Risk Myelodysplastic Syndromes (MDS)/Chronic Myelomonocytic Leukemia (CMML) or Acute Myeloid Leukemia (AML) with 20-30% Marrow Blasts: The Randomized Phase 3 PANTHER Trial (NCT03268954)
Klotho Pathways, Myelination Disorders, Neurodegenerative Diseases, and Epigenetic Drugs
Phase II study of pevonedistat (P) + azacitidine (A) versus A in patients (pts) with higher-risk myelodysplastic syndromes (MDS)/chronic myelomonocytic leukemia (CMML), or low-blast acute myelogenous leukemia (LB AML) (NCT02610777).
A pragmatic patient-reported outcome strategy for rare disease clinical trials: application of the EORTC item library to myelodysplastic syndromes, chronic myelomonocytic leukemia, and acute myeloid leukemia
Effect of CYP3A inhibitors on the pharmacokinetics of pevonedistat in patients with advanced solid tumours
Epigenetic treatment of dermatologic disorders
Pevonedistat, a first-in-class NEDD8-activating enzyme inhibitor, combined with azacitidine in patients with AML
Phase Ib study of pevonedistat, a NEDD8-activating enzyme inhibitor, in combination with docetaxel, carboplatin and paclitaxel, or gemcitabine, in patients with advanced solid tumors
Economic burden of elderly patients with acute myeloid leukemia treated in routine clinical care in the United States
Treatment patterns and health care resource utilization among patients with relapsed/refractory systemic light chain amyloidosis
A New Approach to Treating Neurodegenerative Otologic Disorders
Gut Microbiota and Salivary Diagnostics: The Mouth Is Salivating to Tell Us Something
Epigenetic Treatment of Neurodegenerative Ophthalmic Disorders: An Eye Toward the Future
Microbiota and Neurological Disorders: A Gut Feeling
Epigenetic Treatment of Neurodegenerative Disorders: Alzheimer and Parkinson Diseases
Epigenetic Treatment of Neuropsychiatric Disorders: Autism and Schizophrenia
Epigenetic Treatment of Persistent Viral Infections
Novel Inducers of Fetal Globin Identified through High Throughput Screening (HTS) Are Active In Vivo in Anemic Baboons and Transgenic Mice
The E3 ligase APC/C <sup>Cdh1</sup> promotes ubiquitylation-mediated proteolysis of PAX3 to suppress melanocyte proliferation and melanoma growth
Bioprotective Carnitinoids: Lipoic Acid, Butyrate, and Mitochondria‐Targeting to Treat Radiation Injury: Mitochondrial Drugs Come of Age
MicroRNA let-7 downregulates STAT3 phosphorylation in pancreatic cancer cells by increasing SOCS3 expression