Genome editing is currently being applied to research on cancer, mental health, rare diseases, and many other disease areas.
Basic research exploring human and nonhuman genomes is critical to help scientists understand the basic biology underlying disease, as well as to discover new possible therapeutic targets. Although excitement about the potential for gene therapy has grown tremendously since the discovery of CRISPR, the vast majority of work undertaken by scientists funded by NHGRI or other NIH Institutes takes place in the petri dish and in nonhuman organisms such as mice or zebrafish. Researchers rely on genome editing tools as a way to explore the connection between genotype (genes) and phenotype (traits). A typical study might be to model human disease in mice by deleting or editing certain genes that are thought to contribute to the disease. This approach can help researchers determine if specific changes made to the genome contribute to the disease. It can also lead to the creation of "disease models," or laboratory animals that mimic human diseases and can be studied to test new therapies.
In the clinic, there are proposals to use genome editing as a treatment for disease. Many diseases from cancer to asthma have genetic bases. Through the application of genome editing technologies, physicians might eventually be able to prescribe targeted gene therapy to make corrections to patient genomes and prevent, stop, or reverse disease.