Concurrent Symposia Sessions
Wednesday, September 13, 2017 • Thursday, September 14, 2017
Cell-Based Therapies: Transitioning from the Research Laboratory to the Clinic
Chair: David F. Stroncek, M.D. (CC)
Cell therapies have been used in clinical trials for more than 25 years, but until recently, their effectiveness and clinical application has been limited. The NIH intramural program is well suited to develop and translate novel cell therapies in early phase clinical trials. The results of many of these trials have been promising and some have advanced to later phase clinical trials. This symposium will highlight advances being made by intramural investigators in the fields of cancer immunotherapy, gene therapy, and regenerative medicine and the unique role of the Clinical Center and intramural program in the rapidly emerging field of cell-based therapies.
- Autologous iPS cell based therapy for age-related macular degeneration
Kapil Bharti, Ph.D. (NEI)
- Chimeric antigen receptor (CAR) t cell therapies for hematologic malignancies
James N. Kochenderfer, M.D. (NCI-CCR)
- FARE award winner, Toll-like receptor 2/1 signaling induces human monocytes to differentiate into immunosuppressive macrophage: implications for the treatment of autoimmune and inflammatory diseases
Defne Bayik (NCI-CCR)
- CRISPR gene repair of hematopoietic stem cells in x-linked chronic granulomatous disease
Suk See DeRavin, M.D., Ph.D. (NIAID)
- Role of the cell processing center in developing and improving cell therapies
David F. Stroncek, M.D. (CC)
Chair: Robert S. Adelstein, M.D. (NHLBI)
Developmental biology aims to understand how an organism develops—how a single cell becomes an organized grouping of cells that is then programmed at specific times to become specialized for certain tasks. Genes control much of an organism’s development, but environmental stimuli also play a role, resulting in the complex “nature vs. nurture” paradigm. Intramural Research Program (IRP) investigators seek a deeper understanding of human development and the many disorders and diseases that can occur when normal development is disrupted. The purpose of this symposium is to inform the NIH community of the breadth of research in Developmental Biology being carried at the NIH in different laboratories, using a variety of animal species to model human biology.
Moderator: Brant M. Weinstein, Ph.D. (NICHD)
- A central role for an ancient nutrient stress pathway in the regulation of gametogenesis and early meiotic events in metazoans
Mary A. Lilly, Ph.D. (NICHD)
- Fertilization: the beginning of development
Jurrien Dean, M.D. (NIDDK)
- Morphogenesis without a morphogen: genetic evidence that Shh doesn’t function as a limb morphogen – how does it act?
Susan Mackem, M.D., Ph.D. (NCI-CCR)
- FARE award winner, Investigating molecular mechanisms of microcephaly through mitotic spindle-independent pathways
Todd Schoborg, Ph.D. (NHLBI)
Regulation of mitochondrial retrograde transport in axons
Katie Drerup, Ph.D. (NICHD)
Genotyping and phenotyping
FAES Classrooms 1-4
The genomics era has led to exciting discoveries in human clinical genetics that have greatly improved our understanding of the etiology of rare and common diseases. This symposium will highlight studies incorporating clinical characterization at the NIH Clinical Center with discovery of their genetic etiology. The topics range of topics discussed this symposium including neuromuscular disease, cancer predisposition syndromes, and immunodeficiency are meant to illustrate the breadth and depth of such studies in the intramural research program and highlight opportunities for collaboration.
- Identifying diseases genes and developing treatment for hereditary neuromuscular disease
Kenneth H. Fischbeck, M.D. (NINDS)
- Genotype first, phenotype second: Novel phenotype discovery in the DICER1 syndrome at the Clinical Center in the era of large-scale exome sequencing
Douglas Stewart, M.D. (NCI-DCEG)
- Uncovering etiology and improving patient care through characterization of cancer predisposition syndromes
Sharon A. Savage, M.D. (NCI-DCEG)
- Horror Autoinflammaticus: The expanding spectrum of autoinflammatory disease
Dan Kastner, M.D., Ph.D. (NHGRI)
- What genomics is telling us about the clinical phenotype of neurodegenerative diseases
Bryan Joseph Traynor, M.D., Ph.D., M.M.Sc, M.R.C.P.I (NIA)
- FARE award winner, Mutations in GIMAP5 lead to a novel mendelian disease of autoimmunity
Michael Leney-Green, Ph.D. (NIAID)
Neuroscience and compulsive disorders
FAES Classrooms 6-7
- Hunger-driven motivational state completion
Michael Krashes, Ph.D. (NIDDK)
Goal-directed behaviors are sets of motor actions that direct an animal toward an explicit target object, an interaction that promotes individual survival and/or maintains the species. Despite the necessity of motivated behaviors ranging from ingestive to reproductive to aggressive/defensive exploits, an organism can only perform a single action at any given time, highlighting the tremendous flexibility and speed in which the brain can coordinate complex decision-making. Here Dr. Krashes’s team investigates the role distinct levels of satiety play on shaping innate, motivated drive states as well as the subsets of neurons capable of perturbing innate behavioral choice.
- Alcohol drinking: what is driving the higher risk for alcohol abuse?
Veronica A. Alvarez, Ph.D. (NIAAA)
Several factors have been associated with high alcohol drinking in humans. The team lead by Dr. Alvarez is investigating whether similar factors determine vulnerabilities to high alcohol use in rodents and whether there is link and common mechanism underlying these vulnerabilities.
- Role of anterior insular cortex and amygdala circuits in relapse after voluntary abstinence
Yavin Shaham, Ph.D. (NIDA)
Dr. Shaham will describe a new rat model of drug relapse after choice-based voluntary abstinence and present recent findings on the role of D1- and D2-receptor expressing neuronal ensembles in dorsomedial striatum and glutamatergic projections from anterior insular cortex to central amygdala in this new form of relapse.
- Compulsive and impulsive disorders: polar opposites?
Philip Shaw, Ph.D. (NHGRI)
Dr. Shaw will consider the insights that can be gained into compulsivity through considering the neural substrates of childhood obsessive compulsive disorder. The focus will be on contrasts that can be drawn with childhood disorders in which impulsivity is a prominent symptom- such as attention deficit hyperactivity disorder.
- FARE award winner, Elucidating the role of the supramammillary nucleus in motivational processes
Andrew Kesner, B.A. (NIDA)
Diversity of biological insights from single cell analysis across multiple diseases
Co-chairs: Mark R. Cookson, Ph.D. (NIA) and Yong-Chen William Lu, Ph.D. (NCI-CCR)
This symposium will highlight the application of single cell genomics across many different areas of biomedical interest. We will discuss a variety of exciting cellular and genomics techniques used as well as the challenging but informative informatics analyses that can be applied in this area. We hope that the audience will see opportunities to apply single cell techniques to their own research questions.
- Immune networks from single cells and networks to human populations
John S. Tsang, Ph.D., B.A.Sc., M.Math (NIAID)
- Single Cell Analyses of infected T cells for translational approaches in HIV research
Eli Boritz, M.D., Ph.D. (NIAID)
- A Comprehensive Single Cell Map of Mammalian Auditory Sensory Cell Development
Michael Kelly (NICHD)
- Single Cell Analysis identifies Transcriptional events in Acute Myeloid Leukemia
Jamie Diemer (NHGRI)
- FARE award winner, Direct observation of endogenous transcription and splicing dynamics in single human cells
Yihan Wan (NCI)
Chair: Yasmine Belkaid, Ph.D. (NIAID)
Symposia description forthcoming
- Control of tissue immunity and inflammation by the microbiota
Yasmine Belkaid, Ph.D. (NIAID)
- Could microbiome boost cancer immunotherapy?
Marie A. Vetizou, Ph.D. (NCI-CCR)
- Identification of novel eukaryotic viruses from direct sequencing of human patient samples
Julie A. Segre, Ph.D. (NHGRI)
- GI tract microbiome and microbial translocation in progressive SIV infection of nonhuman primates
Jason Brenchley, M.A., Ph.D. (NIAID)
RNA biology and therapeutics
FAES Classrooms 1-4
DNA serves as the repository for genetic information, and proteins are the biochemical machines of the cell. RNA is remarkable, being able to carry out both functions. This biochemical and structural versatility gives rise to diverse cell biological roles ranging from catalysis to the regulation of chromatin structure and gene expression. Because of its many critical functions, RNA has emerged as an important target for therapeutic intervention. In this session, advances in fundamental RNA biology, structure-based engineering, and pharmacological targeting of RNA in the NIH intramural program will be presented.
- Small RNAs guard genome integrity
Astrid D. Haase, M.D., Ph.D. (NIDDK)
- Regulating gene expression through RNA surveillance
Sandra L. Wolin, M.D., Ph.D. (NCI-CCR)
- Making RNA shine: structure and engineering of fluorescent RNA tags for live cell imaging
Adrian Ferré-D’Amaré, Ph.D. (NHLBI)
- Targeting structurally and functionally diverse nucleic acids with drug-like small molecules
Jay Schneekloth, Ph.D. (NCI-CCR)
- FARE award winner, Grc3 programs the essential endoribonuclease Las1 for specific RNA cleavage
Monica C. Pillon, Ph.D. (NIEHS)