Development and Clinical Applications of Nucleic Acid Therapeutics
Identifieur interne : 000B75 ( Istex/Corpus ); précédent : 000B74; suivant : 000B76Development and Clinical Applications of Nucleic Acid Therapeutics
Auteurs : Veenu Aishwarya ; Anna Kalota ; Alan M. GewirtzSource :
- Advances in Polymer Science [ 0065-3195 ]
Abstract
Abstract: In the past decade, our new understanding of post-transcriptional gene silencing (PTGS) has opened up new vistas that can help us to decipher the function of genes and thus to unravel the basic mechanistic principles and genetic networks in biology. This enormous potential of PTGS to selectively turn off genes either by antisense or RNA interference pathways has made a huge impact on research in basic science. Further, we can apply this knowledge and new set of rules in biomedical research for the development of more potent therapeutics. With a plethora of advantages of PTGS, there are still certain obstacles that need to be addressed to enable translation of this technology to the clinic. This article will focus on a brief history of the discovery and evolution of PTGS and its potential as a technology. We will also discuss the importance of target availability and some strategies for identification of the target sequence. Further, we will give an overview of some past experiences in the development of this technology in the clinic for cancer therapy, with a recent update on clinical development and trials on nucleic acid therapeutics.
Url:
DOI: 10.1007/12_2011_146
Links to Exploration step
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<Para>In the past decade, our new understanding of post-transcriptional gene silencing (PTGS) has opened up new vistas that can help us to decipher the function of genes and thus to unravel the basic mechanistic principles and genetic networks in biology. This enormous potential of PTGS to selectively turn off genes either by antisense or RNA interference pathways has made a huge impact on research in basic science. Further, we can apply this knowledge and new set of rules in biomedical research for the development of more potent therapeutics. With a plethora of advantages of PTGS, there are still certain obstacles that need to be addressed to enable translation of this technology to the clinic. This article will focus on a brief history of the discovery and evolution of PTGS and its potential as a technology. We will also discuss the importance of target availability and some strategies for identification of the target sequence. Further, we will give an overview of some past experiences in the development of this technology in the clinic for cancer therapy, with a recent update on clinical development and trials on nucleic acid therapeutics.</Para>
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<Keyword>Post-transcriptional gene silencing</Keyword>
<Keyword>RNA interference</Keyword>
<Keyword>RNase H</Keyword>
<Keyword>siRNA</Keyword>
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<ArticleNote Type="Misc"><SimplePara>Dedicated to the memory of our mentor and a visionary – Dr. Alan M. Gewirtz.</SimplePara>
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<mods version="3.6"><titleInfo lang="en"><title>Development and Clinical Applications of Nucleic Acid Therapeutics</title>
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<name type="personal" displayLabel="corresp"><namePart type="given">Veenu</namePart>
<namePart type="family">Aishwarya</namePart>
<affiliation>Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania School of Medicine, Philadelphia, PA, USA</affiliation>
<affiliation>E-mail: veenu@mail.med.upenn.edu</affiliation>
<role><roleTerm type="text">author</roleTerm>
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<name type="personal"><namePart type="given">Anna</namePart>
<namePart type="family">Kalota</namePart>
<affiliation>Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania School of Medicine, Philadelphia, PA, USA</affiliation>
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<name type="personal"><namePart type="given">Alan</namePart>
<namePart type="given">M.</namePart>
<namePart type="family">Gewirtz</namePart>
<affiliation>Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania School of Medicine, Philadelphia, PA, USA</affiliation>
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<abstract lang="en">Abstract: In the past decade, our new understanding of post-transcriptional gene silencing (PTGS) has opened up new vistas that can help us to decipher the function of genes and thus to unravel the basic mechanistic principles and genetic networks in biology. This enormous potential of PTGS to selectively turn off genes either by antisense or RNA interference pathways has made a huge impact on research in basic science. Further, we can apply this knowledge and new set of rules in biomedical research for the development of more potent therapeutics. With a plethora of advantages of PTGS, there are still certain obstacles that need to be addressed to enable translation of this technology to the clinic. This article will focus on a brief history of the discovery and evolution of PTGS and its potential as a technology. We will also discuss the importance of target availability and some strategies for identification of the target sequence. Further, we will give an overview of some past experiences in the development of this technology in the clinic for cancer therapy, with a recent update on clinical development and trials on nucleic acid therapeutics.</abstract>
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<topic>Antisense</topic>
<topic>Cancer therapy</topic>
<topic>Clinical trials</topic>
<topic>Oligodeoxynucleotides</topic>
<topic>Post-transcriptional gene silencing</topic>
<topic>RNA interference</topic>
<topic>RNase H</topic>
<topic>siRNA</topic>
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<name type="personal"><namePart type="given">Akira</namePart>
<namePart type="family">Murakami</namePart>
<affiliation>Dept. Biomolecular Engineering, Kyoto Institute of Technology, Matsugasaki, 606-8585, Kyoto, Japan</affiliation>
<affiliation>E-mail: akiram2@kit.ac.jp</affiliation>
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<identifier type="DOI">10.1007/978-3-642-30463-7</identifier>
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