RNA Therapeutics

This self-contained reference covers the most relevant RNA-based technologies, including ribozymes, RNA apatmers, siRNAs, and mRNA-encoding therapeutic proteins. Innovated nanotechnologies are presented, in addition to manufacturing protocols.

Central to the synthesis of proteins, the performance of catalysis, and many other physiological processes, the aberrant expression of which can be linked to human diseases including cancers, RNA has proven to be key target for therapeutics as well as a tool for therapy. In RNA Therapeutics: Function, Design, and Delivery , expert contributors from a broad spectrum of scientific backgrounds highlight the roles that messenger RNAs and small RNAs can play in biology and medicine. While covering the five major RNA-based drugs, namely the use of ribozymes to cleave and/or correct mRNA transcript, the use of siRNA for targeted silencing of gene transcripts, the use of aptamers, like short RNA molecules, for neutralizing the protein functions, the use mRNA-transfected DCs to activate immune system against tumor cells, as well as the use of RNA to reprogram T and/or DC cell function, this extensive volume brings together the fields of coding (mRNA) and non-coding RNA such as ribozymes, RNAse P, siRNAs, and miRNAs into one convenient source. Written in the highly successful Methods in Molecular Biology ™ series format, the cutting-edge protocol chapters contain introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and practical tips on troubleshooting and avoiding known pitfalls. Also, the book contains several excellent reviews for teaching purposes.

Authoritative and comprehensive, RNA Therapeutics: Function, Design, and Delivery provides key models and tools which will assist researchers in increasing our understanding of RNA functions, modifications, and their involvement in diseases in order to lead to the design of vital new RNA-based therapeutics.

Collects in one single volume the most relevant RNA-based technologies, including ribozymes, RNA aptamers, and siRNAs, and mRNA-encoding therapeutic proteins Features the translation of RNA-based therapies into the clinic Includes techniques to isolate, quantify, and clone small RNAs as well as strategies to overcome siRNA unwanted effects Showcases innovative nanotechnologies for RNA delivery and innovative mRNA transfected-DC vaccines Provides protocols for manufacturing non-MHC restricted T cells Includes supplementary material: sn.pub/extras

Inhalt
RNA Modifications: A Mechanism that Modulates Gene Expression.- Quantitative Analysis of RNA Modifications.- Advances in RNA Sensing by the Immune System: Separation of siRNA Unwanted Effects from RNA Interference.- Progress in siRNA Delivery Using Multifunctional Nanoparticles.- Optimized Protocols for siRNA Delivery into Monocytes and Dendritic Cells.- Systemic Delivery of Synthetic siRNAs.- What Are the Key Targeted Delivery Technologies of siRNA Now?.- Using OligoWalk to Identify Efficient siRNA Sequences.- Jet-Injection of Short Hairpin RNA-Encoding Vectors into Tumor Cells.- Short Hairpin RNA (shRNA): Design, Delivery, and Assessment of Gene Knockdown.- Effective Pol III-Expressed Long Hairpin RNAs Targeted to Multiple Unique Sites of HIV-1.- Functional Studies on RNA-Transfected Cell Microarrays.- Transfection Microarrays for High-Throughput Phenotypic Screening of Genes Involved in Cell Migration.- Intron-Mediated RNA Interference, Intronic MicroRNAs, and Applications.- Inhibition of the microRNA Pathway in Zebrafish by siRNA.- Profiling of miRNA Expression and Prediction of Target Genes.- Small RNA Cloning.- In Situ Hybridization Detection of microRNAs.- Detection and Quantitative Analysis of Small RNAs by PCR.- In Vivo Reprogramming of Human Telomerase Reverse Transcriptase (hTERT) by Trans-Splicing Ribozyme to Target Tumor Cells.- Design and Function of Triplex Hairpin Ribozymes.- Catalytic M1GS RNA as an Antiviral Agent in Animals.- Using Live Cells to Generate Aptamers for Cancer Study.- Primer-Free Aptamer Selection Using a Random DNA Library.- Development of TLR7/8 Small RNA Antagonists.- Modulation of Dendritic Cell Maturation and Function by siRNA-Bearing 5?-Triphosphate.- Immunotherapy of Cancer with Dendritic Cells Loaded with Tumor Antigens andActivated Through mRNA Electroporation.- Non-MHC-Dependent Redirected T Cells Against Tumor Cells.- Overcoming Self-Tolerance to Tumour Cells.- Recent Advances in Hematopoietic Stem Cell Transplantation and Perspectives of RNAi Applications.