A5024156069- Hemoglobinopathies and Related Disorders
- Epigenetics and DNA Methylation
- Blood groups and transfusion
- CAR-T cell therapy research
- Iron Metabolism and Disorders
- Erythrocyte Function and Pathophysiology
- RNA modifications and cancer
- Biomedical Ethics and Regulation
- MicroRNA in disease regulation
- Prenatal Screening and Diagnostics
- CRISPR and Genetic Engineering
- Autoimmune Bullous Skin Diseases
- Cancer-related molecular mechanisms research
- Bioactive Compounds and Antitumor Agents
- Myeloproliferative Neoplasms: Diagnosis and Treatment
- Diabetes and associated disorders
- Platelet Disorders and Treatments
- Pharmacological Effects of Natural Compounds
- Neonatal Health and Biochemistry
- Diabetes Management and Research
- Hemophilia Treatment and Research
- Menstrual Health and Disorders
- Hemostasis and retained surgical items
United States Food and Drug Administration
2016-2024
Center for Biologics Evaluation and Research
2022-2024
Center for Drug Evaluation and Research
2023
National Institute of Diabetes and Digestive and Kidney Diseases
2013-2017
National Institutes of Health
2013-2017
Center for Cancer and Blood Disorders
2014-2016
George Washington University
2016
Children's National
2014-2016
Cancer Research And Biostatistics
2016
Rush University Medical Center
2007
In February 2022, the FDA approved ciltacabtagene autoleucel, a chimeric antigen receptor (CAR) T-cell therapy targeting B-cell maturation antigen, for adult patients with relapsed/refractory multiple myeloma after ≥4 lines of therapy, including an immunomodulatory agent, proteasome inhibitor, and anti-CD38 monoclonal antibody. Approval was based on overall response rate (ORR), complete (CR) rate, duration (DoR) in 97 single-arm, open-label, multicenter phase 2 trial (CARTITUDE-1...
Chronic transfusion therapy (CTT) is indicated for stroke prevention in children with sickle cell anemia (SCA) and complicated by iron overload alloimmunization. CTT performed simple (ST), partial manual exchange (PME), or erythrocytapheresis (RCE). Although small case series have demonstrated RCE combination chelation stabilizes and/or decreases ferritin, there are no reports comparing the effect of ST, PME, on liver concentration (LIC). modality serum ferritin LIC were compared SCA...
In humans, the heterochronic cascade composed of RNA-binding protein LIN28 and its major target, let-7 family microRNAs (miRNAs), is highly regulated during human erythroid ontogeny. Additionally, down-regulation miRNAs in cultured adult CD34(+) cells or over-expression erythrocytes from pediatric patients with HbSS genotype causes increased levels fetal hemoglobin (HbF) range 19-40% total. Therefore, we hypothesized that focused targeting individual miRNA members would exhibit regulatory...
Induction of fetal hemoglobin (HbF) has therapeutic importance for patients with sickle cell disease (SCD) and the beta-thalassemias. It was recently reported that increased expression LIN28 proteins or decreased its target let-7 miRNAs enhances HbF levels in cultured primary human erythroblasts from adult healthy donors. Here LIN28A effects were studied further using erythrocytes peripheral blood progenitor cells pediatric subjects SCD. Transgenic accomplished by lentiviral transduction...
Sickle cell anemia (SCA) is an inherited hemolytic with compensatory reticulocytosis. Recent studies have shown that increased levels of reticulocytosis during infancy are associated hospitalizations for SCA sequelae as well cerebrovascular pathologies. In this study, absolute reticulocyte counts (ARC) measured prior to transfusion were analysed among a cohort 29 pediatric patients receiving chronic therapy (CTT) primary and secondary stroke prevention. A cross-sectional flow cytometric...
Increasing fetal hemoglobin (HbF) levels in adult humans remains an active area hematologic research. Here we explored erythroid-specific LIN28A expression for its effect regulating gamma-globin gene and HbF cultured erythroblasts. For this purpose, lentiviral transduction vectors were produced with driven by promoter regions of the human KLF1 or SPTA1 genes. Transgene a linked puromycin resistance marker was restricted to erythroid lineage as demonstrated selective survival colonies...