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:

  1. 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 site-specific endonucleases to augment homologous recombination.

  2. Immunotherapy for cancer and leukemia by modifying HSC to express T cell receptors and chimeric antigen receptors targeted against tumor-associated antigens.

  3. 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 site-specific endonucleases to augment homologous recombination.

  4. Development of improved gene delivery vectors to achieve specific patterns of transgene expression.

 

Summer Student Presentations: