We have made great strides in our ability to treat disease through genetic and cellular engineering. Viral and non-viral vectors enable us to introduce synthetic genes into cells to reprogram cell functions. A potent example of this is the engineering of T cells to express a chimeric antigen receptor (CAR) that directs the T cells to kill cancer cells. With the development of CRISPR/Cas9 and its derivatives as a gene editing technology, researchers can now modify DNA and RNA at desired locations. Whether by knocking out a gene, turning on or off a gene, or adding or altering specific nucleotide sequences, we can change the blueprint of a cell, and use the technology to discover the functions of any gene or establish new disease treatments.
The Icahn Genomics Institute (IGI) is using CRISPR gene editing to engineer stem cells to create important new models of complex human diseases. This includes using CRISPR to turn pluripotent stem cells into neurons to study synaptic plasticity and its dysfunction in Fragile X syndrome and association with autism. We are also reprogramming cells through CRISPR to generate human pluripotent stem cell models of blood diseases, such as myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML), in order to understand disease mechanisms and develop new therapeutic targets.
Scientists in the IGI are also developing new cell engineering and CRISPR technologies, including technologies to target the expression of therapeutic genes in cell-state dependent manners, and rationally designed CRISPR/Cas9 variants that improve the efficiency of therapeutic applications, such as base editing for the correction of deleterious mutations and treatment of inherited genetic diseases.