Gene Editing and Cell Engineering

The Department of Genetics and Genomic Sciences and the Icahn Genomics Institute are at the forefront of the development and application of precision techniques to alter gene and cell function to advance basic and applied biomedical research.

Recent years have seen great advances in gene editing technologies such as CRISPR/Cas9 and TALEN that allow the precise editing of the genome. This capability provides the opportunity to create more accurate cellular and animal models of disease. CRISPR/Cas9 technology used as a gene and cell therapy tool has the potential to revolutionize medicine as a cure for a range of genetic diseases. More broadly, CRISPR/Cas9 library screening approaches allow systematic investigation of pathogenic mechanisms and potential therapeutic drug discovery for a wide variety of tissues and disease. Alternatively, modified RNA or a variety of viral and non-viral vectors can be used to introduce genes or proteins into cells to reprogram cellular functions.

CRISPR/Cas9 and other gene and cell engineering methods can be applied to both stem cells and somatic cells. For example, induced pluripotent stem cells (iPSCs) can be reprogrammed to develop into multiple tissue types, which can be used to study normal tissue development, disease progression, and tissue regeneration, and for therapeutic drug screening. In combination with gene editing, iPSCs can be used to create patient-specific disease models with which to investigate pathologic mechanisms and responses to drug treatment, and correct genetic defects with the goal of normal tissue regeneration, achieving the goals of truly personalized medicine. Gene editing and cell engineering have exciting applications to the study and treatment of a range of critical human diseases, including cardiovascular, neurodegenerative, hematologic, immune, metabolic, and hereditary diseases, and cancer.