• Hematology and Gene Therapy Program

    The Molecular and Gene Therapy Program focuses on developing genetic approaches to treat inherited genetic diseases and cancer, using gene delivery to study disease pathophysiology, developing novel stable gene delivery vectors, and studying the safety of permanent gene delivery into cells.

    Our goal is to facilitate translation of gene therapy research into clinical trials. Our program develops novel genetic, molecular and cell therapy approaches and delivery systems to treat inherited genetic disorders and utilizes genetic and molecular delivery to study disease pathophysiology.

    Punam Malik, MD - program leader
    Theodosia Kalfa, MD, PhD
    Dao Pan, PhD
    Janos Sumegi, MD, PhD 

  • Fanconi anemia studies.

    click to enlarge

  • Hemoglobinopathies.

    click to enlarge