The Experts below are selected from a list of 114897 Experts worldwide ranked by ideXlab platform
Beverly L. Davidson - One of the best experts on this subject based on the ideXlab platform.
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Recent Advances in RNA Interference Therapeutics for CNS Diseases
Neurotherapeutics, 2013Co-Authors: Pavitra S. Ramachandran, Megan S. Keiser, Beverly L. DavidsonAbstract:Over the last decade, RNA Interference technology has shown therapeutic promise in rodent models of dominantly inherited brain diseases, including those caused by polyglutamine repeat expansions in the coding region of the affected gene. For some of these diseases, proof-of concept studies in model organisms have transitioned to safety testing in larger animal models, such as the nonhuman primate. Here, we review recent progress on RNA Interference-based therapies in various model systems. We also highlight outstanding questions or concerns that have emerged as a result of an improved (and ever advancing) understanding of the technologies employed.
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Current prospects for RNA Interference-based therapies
Nature Reviews Genetics, 2011Co-Authors: Beverly L. Davidson, Paul B. MccrayAbstract:RNA Interference (RNAi) is a powerful approach for reducing expression of endogenously expressed proteins for biological applications, or targeting the expression of pathological proteins for therapy. Several delivery methods are available to achieve RNAi in ex vivo and in vivo settings for therapeutic results. The development of RNAi-based therapeutics has advanced sufficiently to allow human clinical trials to begin. Here we outline the broad range of cell-, tissue- and disease-specific approaches under investigation for RNAi therapeutics. The barriers posed by certain cells and tissues are described, as are issues with off-target silencing. RNA Interference (RNAi) is a powerful approach for reducing expression of endogenously expressed proteins. It is widely used for biological applications and is being harnessed to silence mRNAs encoding pathogenic proteins for therapy. Various methods — including delivering RNA oligonucleotides and expressing RNAi triggers from viral vectors — have been developed for successful RNAi in cell culture and in vivo. Recently, RNAi-based gene silencing approaches have been demonstrated in humans, and ongoing clinical trials hold promise for treating fatal disorders or providing alteRNAtives to traditional small molecule therapies. Here we describe the broad range of approaches to achieve targeted gene silencing for therapy, discuss important considerations when developing RNAi triggers for use in humans, and review the current status of clinical trials. RNA Interference can elicit specific gene silencing and so holds great potential for treating infectious or genetic diseases. Several small-RNA-based therapies have now reached clinical trials, but further work is still needed to improve delivery and efficacy.
Li Jing - One of the best experts on this subject based on the ideXlab platform.
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Design and Application in RNA Interference Library
Journal of Medical Molecular Biology, 2006Co-Authors: Li JingAbstract:The developments in human genomic program have supplied a great deal of primary biological information, but understanding of the functional information remained limited. RNA Interference is a rapid and economical technique for research of gene function. The preparation of RNA Interference library targeting a large number of genes to globally knock down expression of genes on genome-wide scale has become a new approach to study cellular biological behaviors. Recently, many designing strategies based on RNA Interference library have been developed and put into application in genetic screening in some fields. These trials will be beneficial to the study of functional genomics.
Gregory J. Hannon - One of the best experts on this subject based on the ideXlab platform.
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RNA Interference
Nature, 2002Co-Authors: Gregory J. HannonAbstract:A conserved biological response to double-stranded RNA, known variously as RNA Interference (RNAi) or post-transcriptional gene silencing, mediates resistance to both endogenous parasitic and exogenous pathogenic nucleic acids, and regulates the expression of protein-coding genes. RNAi has been cultivated as a means to manipulate gene expression experimentally and to probe gene function on a whole-genome scale.
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RNA Interference in adult mice.
Nature, 2002Co-Authors: Anton P. Mccaffrey, Leonard Meuse, Thu-thao T. Pham, Douglas S. Conklin, Gregory J. Hannon, Mark A. KayAbstract:RNA Interference is an evolutionarily conserved surveillance mechanism that responds to double-stranded RNA by sequence-specific silencing of homologous genes. Here we show that transgene expression can be suppressed in adult mice by synthetic small interfering RNAs and by small-hairpin RNAs transcribed in vivo from DNA templates. We also show the therapeutic potential of this technique by demonstrating effective targeting of a sequence from hepatitis C virus by RNA Interference in vivo.
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RNA Interference : RNA
Nature, 2002Co-Authors: Gregory J. HannonAbstract:A conserved biological response to double-stranded RNA, known variously as RNA Interference (RNAi) or post-transcriptional gene silencing, mediates resistance to both endogenous parasitic and exogenous pathogenic nucleic acids, and regulates the expression of protein-coding genes. RNAi has been cultivated as a means to manipulate gene expression experimentally and to probe gene function on a whole-genome scale.
Vadim Iourgenko - One of the best experts on this subject based on the ideXlab platform.
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RNA Interference technologies and their use in cancer research
Current opinion in oncology, 2007Co-Authors: Alex Gaither, Vadim IourgenkoAbstract:PURPOSE OF REVIEW Recently, RNA Interference has evolved into a powerful research tool to functionally characterize genes. Genome-wide RNA Interference reagents can study the loss-of-function phenotypes of candidate genes in the context of various disease model systems. In this review, we discuss the data from the most recent studies using RNA Interference reagents with a focus on RNA Interference-based genomic screening as a tool to expand our knowledge about the molecular basis of cancer. RECENT FINDINGS Tumorigenesis is the result of the progressive accumulation of mutations in genes controlling cell proliferation and death. Various genes carrying these alterations are known to be directly linked to tumor growth; however, how to translate this knowledge into effective chemotherapeutics, nontoxic to normal cells, is still a subject of intensive research. SUMMARY Loss-of-function studies offer a potential for validation of known and unrecognized tumor-associated targets. RNA Interference-mediated gene knockdown can be exploited to study the reprogrammed circuitry of genes, discover gene interactions restricted to cancer cells and identify mechanisms of chemoresistance in cancer cells. In addition, the simultaneous use of cancer drugs and RNA Interference also provides a paradigm to develop strategies to inactivate essential genes promoting neoplastic growth.
Puthupparampil Scaria - One of the best experts on this subject based on the ideXlab platform.
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Pharmaceutical Prospects for RNA Interference
Pharmaceutical Research, 2004Co-Authors: Raymond M. Schiffelers, Martin C. Woodle, Puthupparampil ScariaAbstract:RNA Interference has proven to be a powerful tool in gene function validation. Recently, the first studies were published reporting a disease-modulating activity of the technique, suggesting a promise for RNA Interference as a novel therapeutic strategy. This review discusses the recent advancements in realizing the clinical utility of RNA-Interference.
