In the last 17 years, I developed gene editing tools to improve cancer immunotherapy or promote safer applications of human hematopoietic stem cell (HSC) gene therapy. I pioneered this field since when the first gene editing enzymes were shown to be potentially useful for therapeutic purposes. In 2012, I published a break-through work where we demonstrated for the first time the possibility to genetically inactivate the T cell receptor in primary T cells for improving safety/efficacy of cancer adoptive immunotherapies. This innovative approach is now widely used in immunotherapy field for generating allo-compatible T cells or to express CAR genes under the TCR promoter. In 2014, I developed the first protocol that allows targeted transgene integration in human HSC capable of long-term multilineage repopulation. My current efforts are aimed to move these advanced genetic engineering strategies towards an effective therapeutic treatment for inherited and acquired hematologic diseases.

Pietro Genovese PhD
Boston Children's Hospital
Boston, Massachusetts
United States
Project Title
Towards clinical testing of epitope editing to enable novel adoptive immunotherapies
Program
Translational Research Program