Can modification of either autologous or allogeneic immune cells allow effective treatment of blood diseases and infection with acceptable rates of toxicity?
Accelerating the research to find suitable viral vectors and delivery systems to inhale gene therapy deeply into the lungs. Distal therapy is important for several fatal lung diseases. This is urgent and critical research.
Studies on protective gene variants as described in NHLBI’s Strategic Visioning Forum are critical and should be one of their main priorities.
There is a need to examine the use of recombinant DNA to the heart for correction of genetic abnormalities or restoration of normal signaling pathways to prevent heart failure. However, gene therapy is a complex process and more studies are needed in which tissue targeting, route of delivery, regulation of target gene expression, therapeutic dose, and identification of robust biomarkers are further investigated.