The purpose of Applications Therapeutic of Ribozymes is to provide an overview of the utility of ribozymes to selectively inhibit the expression of RNA. The ribozyme applications appearing in this book should benefit not only those experienced in ribozymes, but also those applying this ribozyme technology for the first time. It is hoped that a better understanding of the therapeutic application ofribozymes will have a significant impact on human disease in the near future. The field ofribozyme biochemistry has come a long way since the initial observation that RNA is capable of catalysis,...

This book is a collection of preclinical and clinical reports on the appli- cation of gene therapy to human disease. The focus of these studies is on cancer and cardiovascular disease. There are two fundamental technologies for delivering therapeutic genes to diseased ceHs: either viral vectors, as discussed by Dr. Bal- main, or non-viral vector systems, as discussed by Dr. Felgner. The strengths and limitations of each of these delivery systems are charac- terized. The use of a therapeutic gene to treat a disease has taken two general approaches. The first is to introduce anormal (i. e. ,...

The purpose of Applications Therapeutic of Ribozymes is to provide an overview of the utility of ribozymes to selectively inhibit the expression of RNA. The ribozyme applications appearing in this book should benefit not only those experienced in ribozymes, but also those applying this ribozyme technology for the first time. It is hoped that a better understanding of the therapeutic application ofribozymes will have a significant impact on human disease in the near future. The field ofribozyme biochemistry has come a long way since the initial observation that RNA is capable of catalysis,...