Hurdles Associated with the Translational Use of Genetically Modified Cells

Recent advancements in the use of genetically modified hematopoietic stem cells (HSCs) and the emergent use of chimeric antigen receptor (CAR) T-cell immunotherapy has highlighted issues associated with the use of genetically engineered cellular products. This review explores some of the challenges linked with translating the use of genetically modified cells.

The use of genetically modified HSCs for ADA-SCID now has European approval and the U.S. Food and Drug Administration recently approved the use of CAR-T cells for relapsed/refractory B-cell acute lymphoblastic leukemia. Current good manufacturing processes have now been developed for the collection, expansion, storage, modification, and administration of genetically modified cells.

Genetically engineered cells can be used for several therapeutic purposes. However, significant challenges remain in making these cellular therapeutics readily available. A better understanding of this technology along with improvements in the manufacturing process is allowing the translation process to become more standardized.