Overview

Oligonucleotides with base sequences complementary (antisense) to a specific RNA or DNA offer the exciting potential of selectively modulating the expression of an individual gene. Those molecules are gaining interest of the rapid progress in molecular biology and broad scope of potential applications in treating human diseases. However, oligonucleotides diffuse poorly through the biological barriers, including cell membrane. In addition, they are rapidly degraded in vivo by nucleases. Thus, to improve the administration of these compounds is a real challenge that this book will, in part, focus on. Therefore, particular emphasis will be placed on the developments of the chemistry as well as on the formulation and the controlled delivery of these compounds. Background information concerning intracellular trafficking and tissue distribution/pharmacokinetics will also be given. In addition, the therapeutical aspects will also be given consideration.The target audience are researchers working within academia and industry as well as those in management positions in companies that are involved in the field of oligonucleotide therapy.; This book is also vital for postgraduate students who need to be well informed about this currently hot topic.

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9781280066870

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