Goal 3: Advance Translational Research
Integrating New Genomic Discoveries with Genome Editing Towards Personalized Medicine
The human genome is a veritable digital library of information that includes millions of regulatory elements and the expansive classes of long and short noncoding RNAs. These noncoding sequences represent a rich source of sequence variation (eg, SNPs), but the role such sequence variants play in the control of gene expression or noncoding RNA function is poorly understood. Many noncoding sequence variants will prove to be of importance in the pathogenesis of cardiovascular and blood-related diseases as well as patient treatment responses. A major goal should be defining all functional noncoding sequences and variants therein that impact the heart, blood, and vasculature using the latest tools in genomics, informatics, and stem cell biology.