원문정보
초록
영어
Gene therapy products are products in which nucleic acids are used to modify the genetic material of cells. Gene therapy products can be broadly classified on the basis of their delivery system. Means for delivering gene therapy products include viral vectors, nucleic acids(naked DNA), or nucleic acids formulated with agents, such as liposomes, that enhance their ability to penetrate the cell. Current gene therapy is experimental and has not proven very successful in
clinical trials. Little progress has been made since the first gene therapy clinical trial began in 1990. Since gene therapy products contain genetic and other biological materials, many of the safety considerations, issues and principles. In all cases the product should be sufficiently characterized to provide reassurance that the preclinical studies have been conducted with material that is representative of that to be administered to humans in the clinical studies.
The potential impact of any modifications to the manufacturing process and the test material during the development programme, for the extrapolation of the animal findings to humans should be considered.