Gene therapy of hematopoietic stem cells: strategies for improvement.
Gene therapy of hematopoietic stem cells (HSC) is limited by low frequency of the target cells, their quiescent nature, poor engraftment of treated HSC, and lack of a selective growth advantage of genetically modified cells. Lentiviral vectors combined with positive selection strategies using conditional cell-growth switches should allow for improvement.
Hossle
Johann P
JP
Division of Immunology/Hematology/Oncology, University-Children's Hospital Zurich, CH-8032 Zurich, Switzerland.
Seger
Reinhard A
RA
Steinhoff
Dirk
D
D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D005822:Genetic Vectors; D006412:Hematopoietic Stem Cells / Q000502:physiology*; D006801:Humans; D012190:Retroviridae / Q000235:genetics