Biopolymers and cell. 1990. Volume 6. № 1. 12 - 31
V. A. Kordium
POSSIBILITIES OF GENE THERAPY FOR TREATMENT AND PREVENTION OF MASS PATHOLOGIES
Summary
The possibilities of gene therapy for treatment of basic mass human pathologies such as atherosclerosis, malignant neoplasms, infectious diseases and diabetes have been analysed. The gene therapy is subdivided into two basic trends: 1) repair of gene defect; 2) gene compensation of function. The first trend is based on the precision replacement of a defective gene or its fragments and requires obligatory knowledge of lesion details. It is mostly implied in terms of gene therapy but refers almost exclusively to classic hereditary diseases caused by defects in gene structure. The second trend provides for the introduction of gene which should compensate for the reduced or generally altered function under specific regulation. In this case the knowledge of the specific gene defect responsible for the pathology is not obligatory. Since mass pathologies are mostly due to the defects caused by unknown damages in the expression regulation of the intact structural gene, they can be repaired via compensation of gene function. The general scheme of gene therapy of basic mass pathologies and the concrete variants for its elucidation have been proposed. Recent publications on gene therapy of basic mass pathologies have been reviewed.