Nucleic acid therapies applications

Early development of nucleic acid therapy was aimed at genetic deficiency diseases which were amenable to replacement therapy, or correction by administration of the protein products of the defective genes, as described in Chapter 3. The introduction of the normal counterpart of a defective gene into cells of the patient, resulting in the long-term production of the missing protein, appeared as a desirable alternative to the repeated parenteral administration of purified protein (Verma, 1990 Caskey, 1992). However, the current span of nucleic acid-based therapies far exceeds this limited application conceived initially (Riordan and Martin, 1991 Crooke, 1992a). 

Written by international experts knowledgeable about many aspects of nucleic acid-based therapeutics, this book will be an essential guide to aspiring scientists interested in the various aspects of nucleic acid-based therapeutics as well as established scientists in the gene therapy field and related disciplines. This book presents a comprehensive account of the structures and physicochemical properties of gene delivery and expression systems, with emphasis on their in vivo applications for the production of therapeutic... 

Peptide nucleic acid (PNA) is another analogue of RNA and DNA that has been considered as a potential ancestor of present day nucleic acids. In this molecule the natural sugar-phosphate backbone has been replaced by peptide-like linkages [218]. In recent years, novel syntheses of PNA have been reported mainly focused on their application for antisense and antigene therapies [219]. The physical-chemical properties of PNA make it both... 

Ethidium bromide is a common fluorescent stain for nucleic acids, ft is reported to have significant anti-tumor and anti-viral properties. However, its potential applications in human therapy are prevented, due to its mutagenic and carcinogenic activities in model organisms. 

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