Genome Editing & Gene Therapy
Precise and efficient CRISPR genome editing as a curative therapy for genetic disorders
We are in the midst of a revolution in genome editing and CRISPR-Cas9 technology was the spark. With unprecedented rapidity, this technology has provided a straightforward, robust, and specific method for genome editing.
Our research focuses on developing genome editing as curative therapy for genetic diseases. Our lab is particularly interested in applying genome editing for gene therapy of hematopoietic genetic disorders such as severe combined immunodeficiency (SCID).
SCIDs are a set of life threatening genetic diseases in which patients are born with mutations in single genes, and are unable to develop functional immune system. While allogeneic bone marrow transplantation can be curative for these disorders, there remain significant limitations to this approach. We believe that the ultimate cure for these diseases will be transplantation of gene-corrected autologous CD34+ hematopoietic stem and progenitor cells (HSPCs).
To be able to apply this approach in the clinic, we must assure that the genome-editing technology is efficient and safe. Hence, our research focuses on developing an optimized CRISPR- genome editing for robust, site specific and non-toxic functional gene correction in HSPCs. Additional aspect of our research focuses on applying the CRISPR technology to treat malignancies using cancer-immunotherapy.
Our strategy seeks to improve cancer-fighting potency of human T lymphocytes by genetic modification of immune receptors which play a key role during cancer immunity cycle.
Overall, we believe that by advancing CRISPR technology our research will accelerate the bench to bedside path and will help in generating a clear trajectory towards future clinical trials.