Cell and Gene Therapeutics Program
Our Cell and Gene Therapeutics Program focuses on developing and implementing novel gene therapy treatments for childhood cancers and bleeding disorders. Our team is dedicated to translating basic science discoveries into clinical applications. Our program:
- Developed a hematopoietic stem cell-directed lentiviral vector gene transfer strategy that will be used to treat, and possibly cure, hemophilia A.
- Created bio-engineered, protein-based therapeutics for hemophilia A that we expect to be delivered at a cost more affordable to patients and families that cannot afford therapeutics.
- Pioneered the use of drug resistance immunotherapy to treat childhood cancers, and are part of an international clinical trial testing the usefulness of T cells engineered to kill cancer.
- Developed and opened a clinical trial to study the benefit of MSCs in decreasing GVHD in patients following a BMT.
Click the button below to learn more about our program and research.