Alternative viral envelopes for oncoretroviruses to increase gene transfer into hematopoietic stem cells.
The hematopoietic stem cell is the target for gene therapy of human blood disease. Low retroviral receptors for the commonly used vectors and quiescence of hematopoietic stem cells are believed to be major obstacles to the success of gene therapy. The development of new stem cell assays has allowed better understanding of the biology and phenotype of hematopoietic stem cells, leading to selection of highly enriched populations of hematopoietic stem cells. Quantitation of retrovirus receptors on these enriched populations of hematopoietic stem cells has resulted in the identification of subpopulations of cells expressing high levels of retrovirus receptors. New promising retrovirus envelopes are being developed. In this review, we discuss those issues that may help to resolve the problem of low gene transfer efficiency into human hematopoietic stem cells.
Hematopoiesis Section/NHGRI/NIH, Building 49, 49 Convent Drive, Bethesda, MD 20892, USA.
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D000818:Animals; D002469:Cell Separation; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D006412:Hematopoietic Stem Cells / Q000166:cytology / Q000378:metabolism*; D006801:Humans; D066298:In Vitro Techniques; D011991:Receptors, Virus / Q000378:metabolism; D012190:Retroviridae / Q000235:genetics*; D014759:Viral Envelope Proteins / Q000235:genetics
D011991:Receptors, Virus; D014759:Viral Envelope Proteins