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- W2048265510 endingPage "544" @default.
- W2048265510 startingPage "523" @default.
- W2048265510 abstract "Ever since the development of technology allowing the transfer of new genes into eukaryotic cells, the hematopoietic system has been an obvious and desirable target for gene therapy. The last 10 years have witnessed an explosion of interest in this approach to treat human disease, both inherited and acquired, with the initiation of multiple clinical protocols. All gene therapy strategies have two essential technical requirements. These are: (1) the efficient introduction of the relevant genetic material into the target cell and (2) the expression of the transgene at therapeutic levels. Conceptual and technical hurdles involved with these requirements are still the objects of active research. To date, the most widely used and best understood vectors for gene transfer in hematopoietic cells are derived from retroviruses, although they suffer from several limitations. However, as gene transfer mechanisms become more efficient and long-term gene expression is enhanced, the variety of diseases that can be tackled by gene therapy will continue to expand. However, until the problem of delivery and subsequent expression is adequately resolved, gene therapy will not realize its full potential. The first part of this review gives an overview of the gene delivery technology available at present to transfer genetic sequences in human somatic cells. The relevance of the hematopoietic system to the development of gene therapy strategies as well as hematopoietic cell-based gene therapy is discussed in the second part." @default.
- W2048265510 created "2016-06-24" @default.
- W2048265510 creator A5015223856 @default.
- W2048265510 creator A5039639102 @default.
- W2048265510 creator A5054443215 @default.
- W2048265510 date "2001-04-01" @default.
- W2048265510 modified "2023-10-14" @default.
- W2048265510 title "Gene therapy: principles and applications to hematopoietic cells" @default.
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