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Advances in RNAi--A Global Technology Assessment (Technical Insights)Published by: Frost & Sullivan Published: Nov. 19, 2004 Table of Contents1. Executive Summary 1. Introduction 1. RNAi Technology in Brief 2. Overview of the RNAi Sector 2. Scope and Methodology 1. Research Scope 2. Research Methodology 2. Research Developments in RNAi Design 1. Research in the Private Sector 1. The Silencer Effect (USA) 2. DNA-Based siRNA Constructs (USA) 3. Novel RNAi Expression Cassettes (USA) 4. Increase RNAi Stability by Chemical Modifications (Belgium) 2. Research in the Public Sector 1. Inhibit More Genes Simultaneously (USA) 2. Take the siDirect Approach (Japan) 3. UMMS Provides Exclusive RNAi Technologies (USA) 3. Research Developments in RNAi Delivery 1. Research in the Private Sector 1. Explore Electroporation for Efficient RNAi Delivery (Germany) 2. Mouse Musings-Development of RNAi Knockdown in Mice (Germany) 3. Novel Lipid and Polymer-Based Transfection Reagents (USA) 4. Adenoviral siRNA Library (Belgium) 5. Electroporation for Vascular Gene Therapy (USA) 2. Research in the Public Sector 1. Research Liposome Technology as Delivery Vehicles (Netherlands) 2. Nonviral Vectors and Formulations (France) 4. Research Developments in RNAi Applications 1. Research in the Private Sector 1. RNAi Candidate for Liver Cancer (Germany) 2. Treat AMD and DR with RNAi (USA) 3. C. elegans as an Animal Model for Human Diseases (Belgium) 4. RNAi for Nonhuman Therapeutics (USA) 5. Novel Nucleotide Chemistry enhances siRNA Therapeutics (Denmark) 6. Genome Wide siRNA Screening (Germany) 7. siRNA-Based SARS Virus Inhibitors (USA) 8. siRNA Agent Slows Macular Degeneration In Animals (USA) 2. Research in the Public Sector 1. TargeTran siRNA Nanoparticle Technology (USA) 2. RNAi Gene Therapy for HIV Infection (USA) 5. RNAi Sector--Technology Assessment 1. Sector Trend Analysis 1. Technology Trends 2. Corporate Collaborations (Part I) 3. Corporate Collaborations (Part II) 2. RNAi Technology Drivers and Challenges 1. Technology Drivers 2. Technology Challenges 6. Key Contacts and Noteworthy Patents 1. Key Contacts 1. Industry Contacts 2. University Contacts 2. Important Patents 1. Patents Part I 2. Patents Part II 7. Frost & Sullivan 2004 Science and Technology Awards 1. Excellence in Research 1. Award Description 2. Award Recipient 2. Excellence in Technology 1. Award Description 2. Award Recipient 3. Technology Innovation Award 1. Award Description 2. Award Recipient 8. Relevant Decision Support Database (DSD) Tables 1. Healthcare Expenditure 1. Government Healthcare Expenditure (1999 to 2006) 2. Private Healthcare Expenditure (1999 to 2006) 3. Total Healthcare Expenditure (1999 to 2006) 4. Percentage Healthcare Expenditure (1999 to 2006) 5. Per Capita Healthcare Expenditure (1999 to 2006) 2. Pharma R & D Expenditure 1. Pharma R & D Expenditure (1999 to 2006) 2. Percentage Pharma R&D Expenditure (1999 to 2006) 3. Cancer Incidence and Mortality 1. Breast Cancer Incidence (1999 to 2006) 2. Breast Cancer Mortality (1999 to 2006) 3. Liver Cancer Incidence (1999 to 2006) 4. Liver Cancer Mortality (1999 to 2006) 4. Cataract and Diabetes 1. Cataract Blindness (1999 to 2006) 2. Cataract Surgeries (1999 to 2006) 3. Diabetes Incidence (1999 to 2006) 4. Diabetes Prevalence (1999 to 2006) AbstractTransfecting RNAi into Target Cells Proves to be a Significant ChallengeDelivering or transfecting ribonucleic acid interference (RNAi) into mammalian cells is one of the biggest challenges facing researchers. In addition, a tedious optimization process is a prerequisite for the transfection of standard cell lines with commonly used lipofection reagents. Successful delivery of RNAi depends on a few key factors such as the target cell line, RNAi concentration, the ratio of RNAi to the transfection reagent, cell confluence during transfection, and incubation time. Another challenge lies in improving the target specificity of RNAi. In this regard, a well designed RNAi has greater chances of success in targeting the messenger RNA (mRNA) of interest and minimizing off-target effects. In order to test and compare RNAi sequences, researchers need to monitor and optimize RNAi purity and integrity, its uptake, and cell viability. This research analyzes emerging technologies and applications in RNAi design and delivery. Technological trends, drivers, challenges, and recent developments assist in creating effective marketing and production strategies. Participants can identify potential collaborators, stay ahead of the competition, and keep abreast of critical developments in their industry. Failure of Related Researches and Technologies Sparks Interest in RNAi Projects After disappointing results from genomic projects involving antisense technologies and ribozyme projects for gene therapy, researchers are looking at RNAi for a more positive feedback. Since its discovery in 1998, the number of published research papers pertaining to RNAi has skyrocketed from 15 to nearly 1,000 in 2003. "The potential this technology holds is amazing, as it provides a new approach to drug discovery, validation, and delivery, amongst other advantages," says the analyst. Given its recent successes in attacking viruses such as HIV, and hepatitis B and C, RNAi holds immense potential for quicker drug delivery. RNAi’s reach is expected to expand in the future, as it attains the capabilities for treating renal and metabolic disorders, and diseases such as cancers and those pertaining to the central nervous system. RNAi Moves from the Realms of High Throughput Screening to Therapeutics While RNAi expertise, at present, lies primarily in high-throughput screening (HTS), the technology is set to begin clinical trials and RNAi-based drugs might be a reality by 2010. RNAi therapies that are likely to reach humans first are those targeting macular degeneration, a leading cause of blindness. "Caused by over expression of vascular endothelial growth factor (VEGF) in cells in the back of the eye, the disease has several attributes that make it an ideal RNAi showcase," explains the analyst.
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