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Critical Reviews™ in Eukaryotic Gene Expression
Fator do impacto: 1.841 FI de cinco anos: 1.927 SJR: 0.649 SNIP: 0.516 CiteScore™: 1.96

ISSN Imprimir: 1045-4403
ISSN On-line: 2162-6502

Critical Reviews™ in Eukaryotic Gene Expression

DOI: 10.1615/CritRevEukarGeneExpr.v6.i1.30
pages 29-57

Gene Therapy: Basic Concepts and Recent Advances

Deborah L. Sokol
Departments of Pathology and Laboratory Medicine,University of Pennsylvania School of Medicine, Philadelphia, PA 19104
Alan M. Gewirtz
Hematology/Oncology, University of Pennsylvania School of Medicine


After years of preliminary in vitro and in vivo modeling, the first human gene therapy clinical trial was initiated ∼5 years ago. The experience gained from this trial, and the others that have come after it, has shown that effective and efficient human gene therapy remains a tantalizing but elusive goal. Nevertheless, interest in this area of therapeutics continues to grow and over 100 gene therapy protocols have now been submitted to the Recombinant DNA Advisory Committee ("RAC") of the National Institutes of Health for approval. The goal of these protocols vary, but include correction of inherited gene defects, engineering new traits into cells such as diminished chemosensitivity in the case of bone marrow cells, or, in the case of tumor cells, expression of new immunophenotypes. Marking cells with a gene whose function can easily be detected, thereby rendering the cell traceable in vitro and in vivo is also the goal of several clinical trials. At the same time, different means of gene transfer are been investigated for the efficiency with which they deliver "therapeutic nucleotides" into their cellular targets. In this review, we recount some of the major developments that have occurred in these various areas and discuss their impact on progess in the field.