Target Specificity is Primary Focus of Research in RNAi Technologies.PALO ALTO Palo Alto, city, California Palo Alto (păl`ō ăl`tō), city (1990 pop. 55,900), Santa Clara co., W Calif.; inc. 1894. Although primarily residential, Palo Alto has aerospace, electronics, and advanced research industries. , Calif. -- While ribonucleic acid Ribonucleic acid (RNA) One of the two major classes of nucleic acid, mainly involved in translating into proteins the genetic information that is carried in deoxyribonucleic acid (DNA). interference (RNAi) offers a fresh approach to drug discovery, validation, and delivery, current research efforts are geared toward improving target specificity. In this regard, scientists are giving top priority to finding the most efficient means to deliver or transfect RNAi into mammalian target genes. Successful delivery of RNAi depends on a few key factors such as the target cell line, RNAi concentration, ratio of RNAi to the transfection trans·fec·tion n. Infection of a bacterium or cell with DNA or RNA isolated from a bacteriophage or from an animal or a plant virus, resulting in replication of the complete virus. reagent, cell confluence during transfection, and incubation time. "Determining the most effective snippet A small amount of something. In the computer field, it often refers to a small piece of program code. of RNA RNA: see nucleic acid. RNA in full ribonucleic acid One of the two main types of nucleic acid (the other being DNA), which functions in cellular protein synthesis in all living cells and replaces DNA as the carrier of genetic for each gene of interest usually requires testing more than three to four different RNAi sequences," says Frost & Sullivan Industry Analyst Giridhar Rao. "To test and compare any given RNAi sequence, researchers need to monitor and optimize RNAi purity, integrity, uptake, and cell viability." Off-target effects are likely to occur when the sense strand In genetics, a sense strand is the strand of DNA transcribed into mRNA during transcription. The immediate product of this transcription is called an (initial) RNA transcript, which is the same as the sense strand except the nucleotides are RNA instead of DNA. of the RNAi fails to guide the gene silencing process properly. Moreover, potential cross-hybridization with mismatched sequences reduces the specificity of RNAi. "The design of an RNAi plays a vital role in determining the success of RNAi experiments," observes Rao. "A well-designed RNAi is more effective in gene silencing and has better chances of targeting the messenger RNA mes·sen·ger RNA n. See mRNA. and minimizing off-target effects." Studies have already shown that select RNAi molecules specifically target and demonstrate effective and sustained reduction of the alpha-synuclein gene expression. Pre-clinical results from these studies forecast the development of RNAi therapeutics to treat Parkinson's disease Parkinson's disease or Parkinsonism, degenerative brain disorder first described by the English surgeon James Parkinson in 1817. When there is no known cause, the disease usually appears after age 40 and is referred to as Parkinson's disease. . The susceptibility of certain RNAi molecules to rapid degradation by nucleases in serum and other body fluids is a concern for most researchers since stability is imperative for success in therapeutic applications. "The key is to attain optimal stability," says Rao. "Very high stability could cause lodging of residual RNAi in the body and this can lead to increased toxicity." In recent studies, RNAi molecules were shown to be stable, with a long half-life, in both serum and brain extracts. Chemical modifications that increase RNAi stability were also successfully developed and tested. Industry and academic research in the RNAi sector continues to grow. Since its discovery in 1998, the number of published research papers pertaining to RNAi has increased from 15 to 1,000 in 2003. After recent successes using RNAi to attack viruses such as HIV HIV (Human Immunodeficiency Virus), either of two closely related retroviruses that invade T-helper lymphocytes and are responsible for AIDS. There are two types of HIV: HIV-1 and HIV-2. HIV-1 is responsible for the vast majority of AIDS in the United States. , and hepatitis B Hepatitis B Definition Hepatitis B is a potentially serious form of liver inflammation due to infection by the hepatitis B virus (HBV). It occurs in both rapidly developing (acute) and long-lasting (chronic) forms, and is one of the most common chronic and C, the technology shows further potential for treating renal and metabolic disorders, cancers, and even diseases of the central nervous system. Having proven itself in high-throughput screening High-throughput screening (HTS), is a method for scientific experimentation especially used in drug discovery and relevant to the fields of biology and chemistry. Purpose and method , RNAi drugs might be a reality by 2010. The RNAi therapy that is likely reach humans first is those targeting macular degeneration macular degeneration, eye disorder causing loss of central vision. The affected area, the macula, lies at the back of the retina and is the part that produces the sharpest vision. , a leading cause of blindness. "Though RNAi looks set to dominate drug validation and other genomic research, standardization is essential for it to leave the realms of the lab and gain acceptance as a mature technology," concludes Rao. If you are interested in a virtual brochure, which provides manufacturers, end-users and other industry participants an overview of the latest analysis of the Advances in RNAi - A Global Technology Assessment, then send an email to Danielle White Danielle Marie White (born February 2, 1992 in Cincinnati, Ohio) is an American singer who was raised in Bradenton, Florida. In 2003, White appeared on the reality television series American Juniors - Corporate Communications at dwhite@frost.com with the following information: your full name, company name, title, telephone number, fax number, and email. Upon receipt of the above information, an overview will be sent to you via e-mail. Technical Insights (TI) is an international technology analysis business that produces a variety of technical news alerts, newsletters, and research services. Advances in RNAi - A Global Technology Assessment, part of the TI Healthcare Vertical Subscription Service, evaluate emerging technologies and applications in RNAi design and delivery. In addition to analyzing the important drivers and challenges facing RNAi technologies, the study also examines the impact of cutting-edge research and development in this field. Executive summaries and interviews are available to the press. Frost & Sullivan, a global growth consulting company, has been partnering with clients to support the development of innovative strategies for more than 40 years. Its advisory expertise integrates growth consulting, growth partnership services and corporate management training to identify and develop opportunities for clients. While serving clientele ranging from virtually every Global 1000 to emerging companies, Frost & Sullivan's comprehensive industry coverage includes a global perspective of all vertical and horizontal industries. 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Advances in RNAi - A Global Technology Assessment
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Keywords in this release: ribonucleic acid interference, RNAi, mammalian target genes, target specificity, transfection, gene silencing, messenger RNA, alpha-synuclein gene expression, Parkinson's disease, RNAi molecules, RNAi stability, high-throughput screening, macular degeneration |
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