Advances in Gene Therapy for Hematologic Disease and Considerations for Transfusion Medicine

Abstract As the list of regulatory agency-approved gene therapies grows, these products are now in the therapeutic spotlight with the potential to cure or dramatically alleviate several benign and malignant hematologic diseases. The mechanisms for gene manipulation are diverse, and include the use of a variety of cell sources and both viral vector- and nuclease-based targeted approaches. Gene editing has also reached the realm of blood component therapy and testing, where cultured products are being developed to improve transfusion support for individuals with rare blood types. In this review, we summarize the milestones in the development of gene therapies for hematologic diseases, mechanisms for gene manipulation, and implications for transfusion medicine and blood centers as these therapies continue to advance and grow.