Current Stem Cell Reports

, Volume 4, Issue 1, pp 39–45 | Cite as

Hurdles Associated with the Translational Use of Genetically Modified Cells

  • Sunil S. Raikar
  • H. Trent Spencer
Stem Cells: Policies from the Bench to the Clinic (HT Spencer, Section Editor)
Part of the following topical collections:
  1. Topical Collection on Stem Cells: Policies from the Bench to the Clinic


Purpose of Review

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.

Recent Findings

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.


Lentiviral vector Transduction of hematopoietic stem cells Chimeric antigen receptor Genetic engineering 


Compliance with Ethical Standards

Conflict of Interest

Sunil S. Raikar and H. Trent Spencer declare that they have no conflict of interest.

Human and Animal Rights and Informed Consent

This article does not contain any studies with human or animal subjects performed by any of the authors.


Papers of particular interest, published recently, have been highlighted as: • Of importance •• Of major importance

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Copyright information

© Springer International Publishing AG, part of Springer Nature 2018

Authors and Affiliations

  1. 1.Cell and Gene Therapy Program, Department of Pediatrics, Aflac Cancer and Blood Disorders Center, Emory Children’s CenterEmory University School of MedicineAtlantaUSA

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