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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.
FacultyPunam Malik, MD - program leaderTheodosia Kalfa, MD, PhDDao Pan, PhDJanos Sumegi, MD, PhD
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Fanconi anemia studies
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