Research

Research in the Cell Biology Section spans the basic sciences, clinical trials, and epidemiology. Bench work involves methods of cell and molecular biology, immunology, and virology. Blood cell production in healthy individuals and especially in patients with bone marrow failure is the main theme. Advanced techniques most recently include single cell RNAseq, CRISPR-Cas9 gene editing, multicolored bar coded flow cytometry, and SomaLogic deep proteomics. Use of patient samples is encouraged, as the Hematology Branch clinic for patients with aplastic anemia, paroxysmal nocturnal hemoglobinuria (PNH), myelodysplastic syndromes (MDS), large granular lymphocytosis (LGL), and constitutional marrow failure is one of the largest in the world. Results in the laboratory have had real impact on the care of patients with these life-threatening illnesses, and access to patient samples over time and with therapeutic perturbation makes the laboratory strategies both innovative and concrete.

Some Current Laboratory Projects

Single cell RNAseq of normal and diseased bone marrow stem and progenitor cells

Gene editing in human cell lines of genes that affect the epigenome

RNAseq and gene editing applied to PNH cells

Murine models of immune mediated marrow failure, potential therapies and mechanisms

Sex hormone action on human marrow cells

Some Current and Planned Clinical Protocals

Eltrombopag in marrow failure, including treatment-naïve SAA, MAA, and MDS

Campath in LGL

Rapamycin to prevent relapse in AA

Sex hormone modulation of telomeres in patients with telomeropathies

Organization and Mentoring

The Cell Biology Section is large and has a nucleus of expert senior staff to provide training and supervision in a wide variety of advanced techniques. The Section is part of the Hematology Branch, and there is close interaction among all the Branch’s members, including sharing of resources and expertise. There is an active program of lectures and group meetings. In addition to knowledge acquisition, clear thinking and crisp verbal and written expression are priorities for development in trainees.

Dr. Young’s former trainees are leaders in hematology in the United States, Asia, Europe, and South America.