Abstract
Hematopoietic stem cell (HSC) gene therapy remains a highly attractive treatment option for many disorders, including hematologic conditions, immunodeficiencies including human immunodeficiency virus (HIV)/acquired immune deficiency syndrome (AIDS), and other genetic disorders such as lysosomal storage diseases. In this review, we discuss the successes, side-effects and limitations of current gene therapy protocols. In addition, we describe the opportunities presented by implementing ex vivo expansion of gene-modified HSC, as well as summarize the most promising ex vivo expansion techniques currently available. We conclude by discussing how some of the current limitations of HSC gene therapy could be overcome by combining novel HSC expansion strategies with gene therapy.
Acknowledgements
This work was supported in part by NIH grants AI080326, DK56465, CA114218, HL084345, HL03644, HL092554, and HL098489. We thank Bonnie Larson and Helen Crawford for help in preparing the manuscript. H.P. Kiem is a Markey Molecular Medicine Investigator and the recipient of the José Carreras/E. Donnall Thomas Endowed Chair for Cancer Research.”
Disclosure of interests: The authors have no competing financial interests to disclose.