Punam Malik, MD
Disclosures: Paid consultant-Aruvant Sciences - 12/06/2021


Dr. Malik is a physician-scientist, who has worked on hematological disorders for the last 20 years. Clinically, she takes care of children with inherited and acquired blood disorders. Her research interests lie in studying the genetic therapies for hematopoietic stem cell disorders with a focus on hemoglobinopathies. She has been studying gene transfer into murine and human hematopoietic stem cells (HSCs) using different viral vector platforms such as -retrovirus (RV), lentivirus (LV), adeno-associated virus (AAV) and foamy virus (FV) vectors, mechanisms by which gene transfer affects HSC long term engraftment potential, and translation of genetic therapies for inherited hematological disorders such as XSCID and leukocyte adhesion deficiency, hemophagocytic lympho-histiocytosis, beta-thalassemia and sickle cell anemia (SCA). She initiated lentiviral gene therapy for SCA from the bench to the clinic. More recently, she has embarked on gene editing approaches for genetic therapies of hematopoietic disorders, with a focus on tailoring the choice of DNA-repair after a site-specific break by Cas9, with a goal of enhancing homology directed repair and reducing the error-prone ‘end joining’ specifically at the Cas9 cut sites. She is currently a Professor of Pediatrics, the Marjorie Johnson Chair of Gene and Cell Therapy, and the Director of the Cincinnati Children’s Comprehensive Sickle Cell Center at the Cancer and Blood Disease Institute, Cincinnati Children’s’ Hospital and University of Cincinnati.