title: Hematopoietic stem cell expansion and gene therapy.
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.
authors: Fred Hutchinson Cancer Research Center, Seattle, Washington, USA.
authors: Korashon Lynn
authors: KL
authors: Watts
authors: Jennifer
authors: J
authors: Adair
authors: Hans-Peter
authors: HP
authors: Kiem
mesh_terms: D000818:Animals; D018929:Cell Culture Techniques; D049109:Cell Proliferation; D003131:Combined Modality Therapy; D004195:Disease Models, Animal; D015316:Genetic Therapy; D006402:Hematologic Diseases / Q000235:genetics* / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation; D006412:Hematopoietic Stem Cells / Q000378:metabolism* / Q000473:pathology; D006801:Humans