John J. Rossi – författare

MicroRNAs (miRNA) are tiny bits of genetic material that were unknown nearly 10 years ago but now represent an exciting field of study in biology. Upon their discovery, researchers revealed for the first time a new mechanism by which microRNA can stop the function of messenger RNA (mRNA) by literally cutting it in half, interfering with the normal function of specific messenger RNAs in gene expression. This "expression" of genes that code for essential proteins is essentially what controls whether a cell turns into a liver, lung, or brain cell, for example. Understanding what activates this process - or stops it - is a key to understanding the biological process and builds a foundation for advances in medicine and other fields. This volume in Methods in Enzymology presents valuable methods for studying MicroRNA, with three sections covering identification of MicroRNAs and their targets; MicroRNA expression, maturation and functional analysis; and MicroRNAs and disease.

RNA Therapeutics: The Evolving Landscape of RNA Therapeutics provides a comprehensive overview of RNA therapeutic modalities, from bench-to-bedside, with an emphasis on the increasingly impactful areas of gene therapy, oligonucleotide therapeutics, gene editing and delivery. International leaders in the field examine RNA-based therapeutics tools that have been developed to-date to modulate cellular processes such as transcription, translation and protein function. Approved RNA-based therapies and lessons learned from failed therapies are discussed in-depth, as are evolving advances in RNA biochemical analysis, and similar advances that are enabling clinical application of RNA-based therapies.

Later sections discuss delivery technologies, remaining hurdles in research and translation, the therapy development process from the lab to the clinic, and novel RNA-based therapies currently in development.

Features leading experts in the field of RNA therapeutics, spanning all classes of RNA therapies Provides a detailed examination of approved RNA therapies and lessons learned from failed therapeutics Covers all aspects of therapeutic discovery and preclinical development, as well as clinical translation, manufacturing and regulatory aspects

New Findings Revolutionize Concepts of Gene FunctionEndogenous small RNAs have been found in various organisms, including humans, mice, flies, worms, fungi, and bacteria. Furthermore, it’s been shown that microRNAs acting as cellular rheostats have the ability to modulate gene expression. In higher eukaryotes, microRNAs may regulate as much as 50 percent of gene expression. Regulation of Gene Expression by Small RNAs brings together the pioneering work of researchers who discuss their work involving a wide variety of small RNA regulatory pathways in organisms ranging from bacteria to humans. In addition to exploring the biogenesis and processing of these regulatory RNAs, they also consider the functional importance of these pathways in host organisms. Assisting current and future researchers, this unique groundbreaking work — Provides a suite of cutting-edge resources for the study of microRNA ontology and function Includes a technology guide for those seeking to assay microRNA expression Explores the mechanisms by which microRNAs regulate gene expression in animal cells, including the regulation of gene expression by RNA-mediated transcriptional gene silencing Discusses a fast and low-cost approach for reversing genetic influences in mammals Looks at breakthroughs in the use of microRNA-based therapy for HIV and cancer This volume captures the essence of the breadth and excitement surrounding the newly discovered regulatory roles of small RNAs. The powerful new approach in the study of gene function described in this text is leading to some remarkable findings that have the potential to revolutionize our understanding of genetic function and the treatment of diseases otherwise considered intractable.

New Findings Revolutionize Concepts of Gene FunctionEndogenous small RNAs have been found in various organisms, including humans, mice, flies, worms, fungi, and bacteria. Furthermore, it’s been shown that microRNAs acting as cellular rheostats have the ability to modulate gene expression. In higher eukaryotes, microRNAs may regulate as much as 50 percent of gene expression. Regulation of Gene Expression by Small RNAs brings together the pioneering work of researchers who discuss their work involving a wide variety of small RNA regulatory pathways in organisms ranging from bacteria to humans. In addition to exploring the biogenesis and processing of these regulatory RNAs, they also consider the functional importance of these pathways in host organisms. Assisting current and future researchers, this unique groundbreaking work — Provides a suite of cutting-edge resources for the study of microRNA ontology and function Includes a technology guide for those seeking to assay microRNA expression Explores the mechanisms by which microRNAs regulate gene expression in animal cells, including the regulation of gene expression by RNA-mediated transcriptional gene silencing Discusses a fast and low-cost approach for reversing genetic influences in mammals Looks at breakthroughs in the use of microRNA-based therapy for HIV and cancer This volume captures the essence of the breadth and excitement surrounding the newly discovered regulatory roles of small RNAs. The powerful new approach in the study of gene function described in this text is leading to some remarkable findings that have the potential to revolutionize our understanding of genetic function and the treatment of diseases otherwise considered intractable.

This volume concentrates on the most relevant oligonucleotide research and drug development areas and emphasizes the basic mechanisms and biology of oligonucleotide reagents. Chapters are devoted to RNAi, oligonucleotide delivery, and immunomodulation. Because of the advanced stages of oligonucleotide-based drug development, preclinical and clinical data are also presented by leaders in the field. NOTE: Annals volumes are available for sale as individual books or as a journal. For information on institutional journal subscriptions, please visit www.blackwellpublishing.com/nyas.ACADEMY MEMBERS: Please contact the New York Academy of Sciences directly to place your order (www.nyas.org). Members of the New York Academy of Science receive full-text access to the Annals online and discounts on print volumes. Please visit http://www.nyas.org/MemberCenter/Join.aspx for more information about becoming a member