
About
Saswati Chatterjee is a Professor in the Department of Surgery at Duarte Cancer Center, focusing on groundbreaking research in gene therapy and stem cell biology. Her work leverages recombinant adeno-associated virus (rAAV) vectors for genetic modification of hematopoietic stem cells, targeting diseases like hemophilia A, HIV, cancer, and atherosclerosis.
Research Focus
- Developing rAAV-mediated genome editing of sinusoidal endothelial stem cells for permanent correction of hemophilia A.
- Investigating rAAV vectors for stable genetic modification of non-dividing stem cells in bone marrow and cord blood.
- Evaluating rAAV-based therapies for atherosclerosis using ApoA1 mutants in mouse models.
Key Findings
Her team has demonstrated that rAAV vectors can transduce quiescent CD34+CD38- stem cells, enabling stable gene integration and multi-lineage engraftment in immune-deficient mice. Crucially, rAAV-modified stem cells persist up to six months post-transplant, with evidence of success in serial transplant assays—a hallmark of true hematopoietic stem cell modification.
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