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Gene therapy: applications and progress towards the clinic
Author(s) -
Trent R. J. A.,
Alexander I. E.
Publication year - 2004
Publication title -
internal medicine journal
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.596
H-Index - 70
eISSN - 1445-5994
pISSN - 1444-0903
DOI - 10.1111/j.1445-5994.2004.00708.x
Subject(s) - genetic enhancement , medicine , progenitor cell , cell therapy , gene transfer , clinical trial , disease , cancer therapy , gene , intensive care medicine , bioinformatics , cancer , stem cell , genetics , pathology , biology
Gene therapy was originally conceived as an approach to the treatment of genetic disease, to repair or replace a faulty gene. Subsequently, gene therapy clinical trials have been undertaken for a wide range of conditions, particularly cancer and AIDS. Overall, the results from gene therapy have been disappointing. The reasons include the following: (i) low gene transfer efficiencies and (ii) shortcomings in the identification and manipulation of appropriate target cells, including progenitor cell populations required for the maintenance of long‐term effects. Today, the immense potential of gene therapy remains, but more basic research is required to improve technical aspects of this form of cellular therapy. (Intern Med J 2004; 34: 621−625)