There is a critical need for the establishment of strategies that will determine the efficacy, safety, and toxicity of genome editing techniques specifically in hematologic diseases.
For lung diseases, how can new technologies for nucleic acid delivery and gene editing be promoted?
Can genome engineering be used to correct or alter lung stem/progenitor cells to ameliorate lung disease and promote regeneration?
The goals of Precision Medicine can be achieved if we determine the biological basis of disease-associated variants for NHLBI diseases. Advances in genetic research have yielded hundreds of disease-associated DNA polymorphisms, yet we lack robust methods to experimentally test their functional relevance in human cells. Determining the molecular and cellular basis of human phenotypic variation is one of the great challenges... more »
How can NHLBI harness the power of novel technologies involving nucleic acid delivery and gene editing for clinical therapeutics?