Advanced Delivery and Therapeutic Applications of Rnai

Un libro in lingua di Cheng Kun (EDT) Mahato Ram I. (EDT) edito da John Wiley & Sons Inc, 2013

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RNA interference (RNAi) is a process in living cells whereby small, modified versions of RNA can interfere with the normal expression of DNA, altering which genes are active and by how much. Researchers are using this process to develop technologies for modifying and studying genetic process, with potential uses in medicine, biotechnology, and functional genomics.

Advanced Delivery and Therapeutic Applications of RNAi is a comprehensive guide to RNAi basics, RNAi delivery and therapeutic applications of RNAi, with contributions from an international panel of experts. Contents include:

• design and mechanism of RNAi molecules: chemical synthesized small interfering RNA (siRNA), long double-stranded RNA (dsRNA), and DNA-based (plasmid or viral vector) short hairpin RNA (shRNA)
• mechanisms and barriers to siRNA and shRNA delivery
• subcellular fate and intracellular trafficking of siRNA, shRNA and mRNA
• role of promoters and miRNA backbone for efficient nuclear export of shRNA
• analysis of siRNA delivery using various methodologies
• PK/PD profiles of small RNAs
• minimizing cytotoxicity and off-target effects
• delivery strategies, including bioconjugation, cationic lipids, biomaterials, carbon nanotubes, motor pRNA, peptides and fusion proteins, polycation-modified mesoporous silica nanoparticles, multifunctional nanoparticles, micelles, dendrimers, nanogels, stem cells, viral mediation, local delivery, and co-delivery.
• therapeutic applications of RNAi in various diseases, including cancer, liver diseases, viral infections, ocular diseases, in stem cell research, and in cell and organ transplantation
• microRNAs as therapeutic agents and targets
• preclinical, regulatory, market, and intellectual perspectives of RNAi technologies
• challenges and opportunities in bringing RNAi technologies from bench to clinic

Advanced Delivery and Therapeutic Applications of RNAi presents the essential aspects of developing RNAi therapeutics, and will find a place on the bookshelves of researchers working in RNAi, drug discovery and delivery, molecular biology, biomedical engineering and biomaterials, pharmacology, and biotechnology.

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