Protection and selection for gene therapy in the hematopoietic system.
Hematopoietic stem cell gene therapy is potentially curative for a number of inherited and acquired disorders. However, poor gene transfer and expression in repopulating hematopoietic stem cells attenuate this potential. Here we review potential means of conferring a selective advantage to hematopoietic stem cells and their progeny, and discuss the issues that surround the use of selective advantages in vivo.
Milsom
Michael D
MD
Cancer Research UK Gene Therapy Group, Paterson Institute for Cancer Research, Christie Hospital NHS Trust, Wilmslow Road, Manchester M20 4BX, UK.
Fairbairn
Leslie J
LJ
D018528:ATP-Binding Cassette Transporters / Q000235:genetics / Q000378:metabolism; D000818:Animals; D015254:DNA Modification Methylases / Q000235:genetics / Q000378:metabolism; D004260:DNA Repair / Q000235:genetics / Q000502:physiology; D004351:Drug Resistance / Q000235:genetics / Q000502:physiology; D015316:Genetic Therapy; D006413:Hematopoietic System / Q000378:metabolism*; D018398:Homeodomain Proteins / Q000235:genetics / Q000378:metabolism; D006801:Humans; D013762:Tetrahydrofolate Dehydrogenase / Q000235:genetics / Q000502:physiology; D014157:Transcription Factors
D018528:ATP-Binding Cassette Transporters; C090460:HOXB4 protein, human; D018398:Homeodomain Proteins; D014157:Transcription Factors; D013762:Tetrahydrofolate Dehydrogenase; D015254:DNA Modification Methylases