Concurrent Symposia Sessions

Wednesday, September 12, 2018 • Thursday, September 13, 2018

Wednesday, September 12, 2018

Cell-Based Gene Editing and CRISPR

Chair: Lothar Hennighausen, Ph.D. (NIDDK)

Cas9 RNA-guided endonucleases have been repurposed from CRISPR adaptive immune systems in bacteria and archaea and hold great promise in basic and translational research. NIH researchers have made an impact in the development and application of CRISPR/Cas9 genome-editing technologies to investigate genetic circuitry in mammals and advance therapies for hematologic disease. A potential limitation for the clinical use of CRISPR-based therapeutics is the frequent introduction of large deletions as the result of double stranded DNA break caused by Cas9. Such limitations might be avoided by deaminase base editing, a newer form of genome editing, which directly converts target C•G base pairs to T•A, or target A•T base pairs to G•C, without inducing double-stranded DNA breaks. This symposium will cover a range of topics, from the identification of novel CRISPR-Cas systems to the application of advanced base editing in altering the mammalian genome. It is our goal to highlight innovative research that takes emerging technologies from early stages of development to their application in basic science and to medicine.

• Computational approaches to discover novel CRISPR-Cas systems
  Sergey Shmakov, Ph.D. (NLM/NCBI)
• Mutating the mouse genome using CRISPR/Cas9 genome editing and deaminase base editing
  Chengyu Liu, Ph.D. (NHLBI)
  Hye Kyung Lee, Ph.D. (NIDDK) (FARE awardee) 
• Structural and functional dissection of mammalian Mediator complex using CRISPR/Cas9
  Jens Kalchschmidt (NIAMS)
• The lncRNA NeST regulates Ifng expression by inducing chromatin loop formation
  Franziska Petermann (NIAMS)
• Genome engineering of the primate hematopoietic system to study aging and advanced therapies for hematologic disease
  Kyung-Rok Yu (NHLBI)

Myeloid Malignancies

Co-Chairs: Christopher S. Hourigan, M.D., D.Phil (NHLBI) and Paul Liu, Ph.D. (NHGRI)

Myeloid malignancies, such as myelodysplastic syndromes and acute myeloid leukemia, are rare, often highly fatal, cancers of the blood cells. The ability to repeatedly access the predominant sites of disease (blood and bone marrow) has accelerated scientific research into these conditions, resulting in generation of knowledge that is often also applicable to more common, but harder to study, cancer types. The results of these research efforts have been quickly translated, placing myeloid malignancies at the cutting edge of "precision medicine" efforts. In this session, we will highlight the bedside-to-bench-and-back approach that investigators at the NIH intramural research program are taking to this diverse collection of diseases, focusing particularly on mechanistic understanding of genetic etiology and on immune-based and targeted approaches as novel therapy.

• Dual role of JAK inhibitors for GVHD and myeloid malignancy control after allogeneic hematopoietic stem cell transplantation
  Steven Z. Pavletic, M.D., M.S. (NCI-CCR) 
• Familial platelet disorder with propensity to develop myeloid malignancy: bench to bedside and back again
  Lea Cunningham, M.D. (NCI-CCR) 
• The splicing factor U2AF1 contributes to cancer progression through a non-canonical role in translation regulation
  Daniel R. Larson, Ph.D. (NCI-CCR) 
• Chromosome organization and rearrangements in myeloid malignancies
  Kobi Maman, Ph.D. (NCI-CCR)
• Aberrant clonal hematopoiesis of the erythroid and myeloid lineages in a lentivirally barcoded rhesus macaque
  Xing Fan, M.D. (NHLBI) (FARE awardee) 
• CAR T-cell therapy for AML
  Nirali N. Shah, M.D., MHSc (NCI-CCR)

Vascular Biology

Co-Chairs: Hans Ackerman, M.D., D.Phil. (NIAID) and Manfred Boehm, M.D. (NHLBI)

The vasculature is omnipresent, reaching, and interacting with all other organ systems. Vascular diseases are responsible for more than fifty percent of all deaths worldwide with a financial burden of more than $700 billion per year affecting particular lower and middle income populations. Metabolic alterations, systemic inflammation, and hemodynamic tensions, are some of the most important causes that trigger adaptive remodeling processes which may lead to vascular diseases. This symposium will highlight research conducted at the NIH that is helping to uncover the mechanisms by which acquired and genetic factors contributes to the development of vascular diseases.

• Welcoming remarks 
  Manfred Boehm, M.D. (NHLBI)
• Of mice and men, a translational approach to elastin-mediated vasculopathy
  Beth Kozel, M.D., Ph.D. (NHLBI)
• Assessment of vascular inflammation in large-vessel vasculitis: more than meets the eye
  Peter Grayson, M.D., M.Sc. (NIAMS)
• Unraveling social determinants of cardiovascular health
  Tiffany M. Powell-Wiley, M.D., M.P.H., FAHA (NHLBI/NIMHD)
• Measuring microvascular function in children with cerebral malaria
  Hans Ackerman, M.D., D.Phil. (NIAID)
• Aortic B cells: Identifying antibodies specific for atherosclerosis in mice
  Mark Hutchinson, Ph.D. (NIA) (FARE awardee) 
• Mosquito salivary proteins as modulators of vertebrate vascular biology
  Eric Calvo, Ph.D. (NIAID)

Technology Transfer: Successful Partnerships

Chair: Sabarni K. Chatterjee, Ph.D., M.B.A.

The Technology Transfer session will focus on the role of Technology Transfer in building successful partnerships with the industry and extramural community. Scientists will share stories on successful collaborations between their laboratory and industry partners

1:30 p.m.–2:30 p.m. – Role of technology transfer: who we are and what we do

Speakers:
Michael Salgaller, Ph.D., Technology Transfer Center, NCI
Sue Ano, Ph.D. Office of Technology Transfer, NINDS
Steve Ferguson, M.B.A., Office of Technology Transfer, OD/NIH

2:30 p.m.–3:30 p.m. – Panel on technology transfer success stories: successful collaborations and partnerships 
Moderator: Thomas Stackhouse, Ph.D. Associate Director, TTC, NCI

Panelists:
Mitchell Ho, Ph.D.
James W. Hodge, Ph.D., M.B.A.
Malcolm Smith, M.D., Ph.D.

Thursday, September 13, 2018

Computational Biology, Data Science, and Machine Learning

Co-Chairs: Matthew Guay, Ph.D. (NIBIB) and Eytan Ruppin, M.D., Ph.D. (NCI-CCR)

As technological advances enable the collection of more and more data across biomedical domains, researchers must process and find structure in big datasets in order to make full use of their analytical tools. Now, research groups in several NIH institutes are working to develop and apply new machine learning techniques to challenging data analysis problems. In this symposium, four groups will discuss their biomedical machine learning projects. A fifth presentation will share the results of the NIH Research Festival Hackathon, which brings teams together over the course of three days to tackle computational problems of interest to the NIH IRP.

• DeepLesion: a large-scale lesion image dataset and its applications using deep learning
  Ke Yan (CC)
• Robust prediction of response to immune checkpoint blockade therapy i­n metastatic melanoma
  Noam Auslander (NCI-CCR)
• PubMed and beyond: text mining for biology
  Zhiyong Lu, Ph.D. (NLM/NCBI)
• The NIMH machine learning team
  Francisco Pereira (NIMH)
• Hackathon summary presentations 
  Allissa Dillman, Ph.D. (NLM/NCBI)

High Throughput Sequencing – From Single Cell Sequencing to Changing Patient Care

Co-Chairs: Amy P. Hsu, B.A. (NIAID) and Matthew W. Kelley, Ph.D. (NIDCD)

High-throughput, next-generation sequencing (NGS) has allowed the expansion of nucleic-acid research from single, targeted, gene sequencing and transcript analysis, to in-depth investigation of the transcriptional profiles of single cells, detection and characterization of somatic mutations in cancer, and identification of novel disease genes in patients. This session aims to highlight intramural investigators who are using NGS as an integral part of their research program.

• Single cell RNAseq analysis of murine salivary gland development
  Belinda R. Hauser, Ph.D. (NIDCR)
• Using massively parallel single-nucleus sequencing to map neuronal activity after behavior
  Ariel Levine, M.D., Ph.D. (NINDS)
• Profiling a severe drug hypersensitivity syndrome with single-cell RNA-sequencing
  Keisuke (Chris) Nagao, Ph.D. (NIAMS)
• Discovering the transcriptional regulation of mouse Merkel cell development using FACS-based single cell RNA-seq 
  Lingling Miao, Ph.D. (NIAMS) (FARE awardee)
• ClinOmics – apply genomics to patient care at CCR
  Jun S. Wei, Ph.D. (NCI-CCR)
• Disease gene discovery for neurodevelopmental disorders
  Robert B. Hufnagel, M.D., Ph.D. (NEI) 

Chemical Biology

Co-Chairs: Clifton Barry, Ph.D. (NIAID) and Joel P. Schneider, Ph.D. (NCI-CCR)

Chemistry is a powerful tool to probe biological processes and is the foundation on which many new therapies are derived. This session highlights the breadth of biology-including nucleic acid structure and function, amyloid formation, metastasis, cell transporters and HIV-that are being interrogated by intramural investigators using chemical methods.

• Targeting structurally and functionally diverse nucleic acids with druglike small molecules
  John 'Jay' Schneekloth Jr., Ph.D. (NCI)
• Region-specific beta-sheet conformation during alpha-synuclein aggregation revealed by segmental 13C-labeling and Raman microspectroscopy
  Jennifer Lee, Ph.D. (NHLBI)
• Disassembly of the perinucleolar compartment as a therapeutic approach for metastasis
  Juan Jose Marugan, Ph.D. (NCATS)
• Tracking transporters by STORM
  Amy Hauck Newman, Ph.D. (NIDA)
• Disrupting HIV nucleocapsid protein with mercaptobenzamides
  Daniel Appella, Ph.D. (NIDDK)
• Bright, stable, heptamethine cyanine fluorophores for in vivo cancer imaging
  Michael P. Luciano, Ph.D. (NCI-CCR) (FARE awardee)