We discovered a way to express a gene or protein of interest in cancer cells, but not healthy normal cells. Specifically, we created synthetic (not occurring in nature) introns that we can introduce into any gene of interest, such as a “killer gene,” such that the encoded protein is produced in cancer cells carrying a defined, cancer-causing mutation, but not produced in healthy normal cells that do not carry that specific mutation. These types of cancer-causing mutations are mainly identified in patients with myelodysplastic syndromes, a group of cancers in which immature blood cells in the bone marrow do not mature or become healthy blood cells. We believe that these synthetic introns will enable the development of new cancer therapeutics that are highly specific to cancer cells while sparing healthy cells. We aim to develop this technology to better understand and cure specific blood cancers.
- myelodysplastic syndromes (MDS)