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.
Cancer Research UK Gene Therapy Group, Paterson Institute for Cancer Research, Christie Hospital NHS Trust, Wilmslow Road, Manchester M20 4BX, UK.
Michael D
MD
Milsom
Leslie J
LJ
Fairbairn
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