Abstract
Cell therapies are taking center stage in medicine spanning cancer immunotherapy, stem cell engineering, and regenerative medicine. Recently, the first ex vivo hematopoietic stem cell (HSC) gene therapy for the treatment of adenosine deaminase deficiency-severe combined immunodeficiency (ADA-SCID) received marketing approval.1 By now, the majority of the 150 plus patients who have received HSC gene therapy to treat monogenic diseases have demonstrated clinical benefits.2 Promising clinical outcomes in phase I/II trials based on T cells engineered to express T cell chimeric antigen receptors (CARs)3 against hematological malignancies have also spurred unprecedented interest from pharmaceutical and biotechnology companies.
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