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.
Goal 3: Advance Translational Research
To facilitate innovation and accelerate research translation, knowledge dissemination, and implementation science that enhances public health.
Showing 4 ideas for tag "editing"
87 up votes
18 down votes
For lung diseases, how can new technologies for nucleic acid delivery and gene editing be promoted?
3 up votes
3 down votes
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 »
8 up votes
27 down votes
How can NHLBI harness the power of novel technologies involving nucleic acid delivery and gene editing for clinical therapeutics?
2 up votes
4 down votes