Objective 2: Investigate newly discovered pathobiological mechanisms important to the onset and progression of heart, lung, and blood diseases and sleep disorders

Discovering new pathobiological mechanisms and understanding them is critical to improving HLBS health. Such discoveries and research can potentially lead to clinical and implementation studies, which in turn can inform new therapeutic strategies and clinical practice. Important areas to focus on, with the help of emerging technologies such as molecular imaging and nanotechnology, include mechanisms involved in the pathogenesis of rare and common diseases, disease-related structural and functional changes, and the clinical significance of these changes. In addition, tracking disease onset and progression across the lifespan is essential because it has the potential to inform our understanding of how chronic conditions become acute disease and how early exposures (environmental, etc.) and interventions, including lifestyle changes, impact health.

Envision a future in which we can...
  • Preempt the transition from health to disease and enable disease prevention through an enhanced understanding of the pathobiological mechanisms underpinning the early onset of heart, lung, and blood diseases and sleep disorders.
  • Leverage high-throughput multiomics and other advanced technologies for deep phenotyping in longitudinal cohort studies to define the functional significance of genetic and epigenetic variants on human pathobiology, as well as their impact on clinical outcomes involving heart, lung, and blood diseases and sleep disorders across the lifespan.
  • Gain new insights into modifier genes and molecular pathways that influence the variable clinical manifestations and outcomes of monogenic disorders (e.g., sickle cell disease and cystic fibrosis) and structural defects (e.g., congenital heart disease), thereby catalyzing new tailored therapeutic strategies.
  • Identify commonalities underlying disparate heart, lung, and blood diseases and sleep disorders to understand shared pathways and to identify shared therapeutic targets.
  • Leverage emerging human complex tissue and three-dimensional cellular systems to model the pathobiological processes and to identify targetable molecular/cellular pathways that drive the onset and progression of heart, lung, and blood diseases and sleep disorders.

Overarching Objectives

  1. Understand normal biological function and resilience
  2. Investigate newly discovered pathobiological mechanisms important to the onset and progression of heart, lung, and blood diseases and sleep disorders
  3. Investigate factors that account for differences in health status among populations
  4. Identify factors that account for individual differences in pathobiology and in responses to treatments
  5. Develop and optimize novel diagnostic and therapeutic strategies to prevent, treat, and cure heart, lung, and blood diseases and sleep disorders
  6. Optimize clinical and implementation research to improve health and reduce disease
  7. Leverage emerging opportunities in data science to open new frontiers in research on heart, lung, and blood diseases and sleep disorders
  8. Further develop and sustain a scientific workforce capable of accomplishing NHLBI’s mission