Автор: Alain Rolland, Sean M. Sullivan Название: Pharmaceutical Gene Delivery Systems Издательство: CRC Press Год: 2003 ISBN: 9780824742355; 0824742354 Серия: Drugs and the Pharmaceutical Sciences (Book 131) Язык: English Формат: pdf Размер: 10,1 mb Страниц: 456
Gene therapy is a rapidly advancing field with great potential for the treatment of genetic and acquired systemic diseases. In developing pharmaceutical gene therapies, the disease, the therapeutic genes, and the gene delivery system need to be taken into consideration. The disease areas that have been most investigated in gene therapy to date are cancer and cardiovascular, pulmonary, and infectious diseases. Therapeutic genes for each of these diseases have been identified and are either in the clinic or due to enter the clinic. As future generation products are developed, the search for better therapeutic genes for the diseases currently being treated and genes to treat other diseases will continue. What remains to be identified is the gene delivery system needed to control the spatial and temporal modulation of the gene function. Furthermore, the duration, fidelity, regulation, and level of gene expression will be essential features to control by the design of specific gene expression systems.
This book is divided into the following sections: an introduction providing an overview of gene therapy, including a brief history summarizing the field of plasmid and virus-based gene delivery; a description of the present studies in the clinics covering the highlighted technology for each clinical application; a review of the plasmid-based expression systems; a summary of the present plasmid-based gene delivery technologies; the gene therapy applications, both preclinical and clinical; and new technologies for expansion of the applications to new diseases.