Objective 5: Develop and optimize novel diagnostic and therapeutic strategies to prevent, treat, and cure heart, lung, and blood diseases and sleep disorders

Recent scientific and technological developments offer especially promising opportunities to prevent disease, manage and treat illness, and promote resilience. These wide-ranging developments include new gene editing techniques to safely treat and prevent HLBS diseases and disorders, smart tools that monitor and adjust biological processes, techniques that modulate stem cell or immune system signaling to reduce disease risk, and advances in materials science that could yield vastly improved implant devices. Other technologies and bioengineering developments may further facilitate diagnostic capabilities. New lifestyle interventions may improve the maintenance of continued health and wellness and may enable behavioral modification to prevent disease. Many of these advances, which are the results of past investments in basic research, are crucial to developing and optimizing novel diagnostics and therapeutics strategies.

Envision a future in which we can...
  • Develop surgical grafts (using stem cells, 3D printing, and nanotechnologies) for children born with heart defects, in which the grafts grow and adapt into adulthood, making multiple operations unnecessary.
  • Transform the safety and availability of the nation’s blood supply by using genetically engineered stem cells to eliminate histoincompatible antigens and generate universal donor red blood cells that are available of the shelf by a high-throughput process.
  • Build intelligent health systems for HLBS diseases and disorders that use artificial intelligence and machine learning tools that can screen patients for risk factors for early disease development and that can diagnose and evaluate the effects of interventions and treatments.
  • Create biomimetic platforms that promote lung tissue regrowth, regeneration, repair, and replacement of a patient’s individual lung cells and/or tissues to reduce the burden of underlying lung diseases.

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