The major focus of the research in the Kohn lab is the development and implementation of gene therapy using hematopoietic stem cells (HSC).
Current projects include:
- New approaches to gene therapy for ADA-deficient SCID, including the use of lentiviral and foamy viral vectors for ex vivo gene transfer to HSC, direct in vivo ADA gene delivery and ADA gene correction using zinc finger nucleases to augment homologous recombination.
- Immunotherapy for cancer and leukemia by modifying HSC to express T cell receptors and chimeric antigen receptors targeted against tumor-associated antigens.
- New approaches to gene therapy for sickle cell disease, including the use of lentiviral for ex vivo transfer of an “anti-sickling” beta-globin gene to HSC, and beta-globin gene correction using zinc finger nucleases to augment homologous recombination.
- Development of improved gene delivery vectors to achieve specific patterns of transgene expression.