This volume in the prestigious Methods in Enzymology series discusses methods currently used in preclinical and clinical gene therapy. Subjects covered in this book, such as the use of adeno-associated virus delivery for treatment of Parkinson's disease, are topical and are presented in the methods-oriented style popularized by this series.
This volume in the prestigious Methods in Enzymology series discusses methods currently used in preclinical and clinical gene therapy. Subjects covered in this book, such as the use of adeno-associated virus delivery for treatment of Parkinson's disease, are topical and are presented in the methods-oriented style popularized by this series.

Antisense technology is the ability to manipulate gene expression within mammalian cells providing powerful experimental approaches for the study of gene function and gene regulation. For example, methods that inhibit gene expression permit studies which probe the normal function of a specific product within a cell. Such methodology can be used in many disciplines such as pharmacology, oncology, genetics, cell biology, developmental biology, molecular biology, biochemistry, and neurosciences. This volume will be a truly important tool in biomedical-oriented research.
The critically acclaimed laboratory standard for more than forty years, Methods in Enzymology is one of the most highly respected publications in the field of biochemistry. Since 1955, each volume has been eagerly awaited, frequently consulted, and praised by researchers and reviewers alike. Now with more than 300 volumes (all of them still in print), the series contains much material still relevant today--truly an essential publication for researchers in all fields of life sciences.

A timely primer on the new reality of antisense and RNA inhibition for treating a broad range of diseases. The authors show how antisense oligonucleotides are being designed and studied in relation to hypertension, various cancers, inflammatory bowel disease, brain disorders, the blood-brain barrier, and drug delivery. Highlights include RNA-based therapies for many diseases, up-to-date methods and applications, and insight into the enormous potential to provide a new generation of drugs.

Scientific advances over the past two decades have afforded unprecedented oppor tunities to understand the structure and function of receptors, receptor-ligand interactions, and receptor signaling. The extent ofprogress in this area is underscored by the recent Nobel Prize for Medicine and Physiology to Alfred Gilman and Martin Rodbell, both of whose work in understanding receptorlG-protein interactions has redefined the way in which we think of how hormones and neurochemicals exert their activity on cellular function. This book is replete with examples of current research approaches to help us better understand the cellular roles in which the renin-angiotensin system and the angiotensin receptors participate. Clearly, defining the structure of angiotensin receptor subtypes is an important first step in cJarifying the mechanisms by which these receptors take part in cellular function. However, the chapters within this book range far beyond structural studies and encompass research on tissue specific expression of the angiotensin receptor subtypes, the genetic regulation ofthese receptors, and the unique function ofvarious angiotensin subtypes in different organ systems, such as the brain, the reproductive system, adipose tissue, the heart, and the kidneys."

A timely primer on the new reality of antisense and RNA inhibition for treating a broad range of diseases. The authors show how antisense oligonucleotides are being designed and studied in relation to hypertension, various cancers, inflammatory bowel disease, brain disorders, the blood-brain barrier, and drug delivery. Highlights include RNA-based therapies for many diseases, up-to-date methods and applications, and insight into the enormous potential to provide a new generation of drugs.