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Titolo:
Gene therapy: principles and applications to hematopoietic cells
Autore:
Van Tendeloo, VFI; Van Broeckhoven, C; Berneman, ZN;
Indirizzi:
Univ Antwerp, Univ Antwerp Hosp, Lab Expt Hematol, B-2020 Antwerp, BelgiumUniv Antwerp Antwerp Belgium B-2020 xpt Hematol, B-2020 Antwerp, Belgium Univ Antwerp, Flanders Interuniv Inst Biotechnol, Dept Mol Genet, Mol Genet Lab, B-2020 Antwerp, Belgium Univ Antwerp Antwerp Belgium B-2020 l Genet Lab, B-2020 Antwerp, Belgium
Titolo Testata:
LEUKEMIA
fascicolo: 4, volume: 15, anno: 2001,
pagine: 523 - 544
SICI:
0887-6924(200104)15:4<523:GTPAAT>2.0.ZU;2-A
Fonte:
ISI
Lingua:
ENG
Soggetto:
RECOMBINANT ADENOASSOCIATED VIRUS; EPSTEIN-BARR-VIRUS; PERIPHERAL-BLOOD LYMPHOCYTES; COLONY-STIMULATING FACTOR; IMMUNE-DEFICIENT MICE; MARROW REPOPULATING CELLS; POLYLYSINE-DNA COMPLEXES; LONG-TERM EXPRESSION; POSTTRANSCRIPTIONAL REGULATORY ELEMENT; RETROVIRAL-MEDIATED TRANSDUCTION;
Keywords:
gene therapy; hematopoietic cells; hematopoietic stem cells; T lymphocytes; viral gene transfer; recombinant viral vectors; retrovirus; adenovirus; adeno-associated virus (AAV); lentivirus; herpes simplex virus; nonviral gene transfer; lipofection; receptor-mediated gene transfer; particle-mediated gene transfer; DNA injection; electroporation; extrachromosomal replicating vectors;
Tipo documento:
Review
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
281
Recensione:
Indirizzi per estratti:
Indirizzo: Berneman, ZN Univ Antwerp, Univ Antwerp Hosp, Dept Hematol, Wilrijkstr 10,B-2650 Edegem, Belgium Univ Antwerp Wilrijkstr 10 Edegem Belgium B-2650 em, Belgium
Citazione:
V.F.I. Van Tendeloo et al., "Gene therapy: principles and applications to hematopoietic cells", LEUKEMIA, 15(4), 2001, pp. 523-544

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 targetcell 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.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 06/04/20 alle ore 08:27:28