RNA Interference and Development of New Drugs.
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Accession number;06A0292925
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| Title;RNA Interference and Development of New Drugs. |
| Author;
MORITOYO TAKASHI
(Ehime Univ., JPN)
NOMOTO MASAHIRO
(Ehime Univ., JPN)
NOMOTO MASAHIRO
(Ehime Univ., JPN)
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Journal Title;Neurological Therapeutics
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Journal Code:X0110A
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ISSN:0916-8443
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VOL.23;NO.1;PAGE.37-43(2006)
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| Figure&Table&Reference;FIG.1, REF.36 |
| Pub. Country;Japan |
| Language;Japanese |
| Abstract;RNA interference (RNAi) technology was shown to be useful to silencing gene expression in mammalian cells recently. Short RNAs, called small interfering RNAs (siRNA), made the effect of gene silencing. RNAi is now a powerful tool for gene control with a huge impact on both the basic and clinical research. The field of its contribution toward development of new drugs consists of identification and validation of disease targets for new drugs and development of RNAi itself as therapeutics. Many research groups offer in vitro high-throughput screening of cell lines using a library of siRNAs or shRNAs designed against the human genome, and against potentially druggable targets. For the target-discovery process, it is important to translate in vitro results into animal models. Some companies are trying to developing animal models for in vivo target validation using short hairpin (sh) RNA, expressed from viral or other vectors. On the other hand, siRNA itself can be developed as a drug. The key step for achieving effective RNAi in vivo is delivery to the desired organ and into the target cells, to ensure specificity and adequate dose. Depending on the target cells and tissues, many kinds of approach are tried to introduce RNAi locally or systematically. It will probably need to develop different vectors for different tissues. Safty is another important issue if RNAi is applied for humans, as siRNA and vector-expressed shRNA has potential to produce non-targeted effects and could lead side effects. Virus vectors also have risks of insertional mutagenesis and cancer. The first clinical trial using siRNA was initiated for the treatment of wet age-related macular degeneration (AMD). There are a lot of plans to apply RNAi to the treatment of many disorder. (author abst.) |
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