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Antisense Oligonucleotide Drug Design

Current Pharmaceutical Design, 2004
Maneuvering single gene expression is not only an optimal way to study gene function but also an ambitious goal, which will lead to the treatment of a variety of human diseases whose main pathogenetic event is a genetic alteration. The recent efforts focusing on the genome project have led to array based, high throughput, gene expression analysis ...
SCHIAVONE, NICOLA   +3 more
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Pharmacology of Antisense Drugs

Annual Review of Pharmacology and Toxicology, 2017
Recent studies have led to a greater appreciation of the diverse roles RNAs play in maintaining normal cellular function and how they contribute to disease pathology, broadening the number of potential therapeutic targets. Antisense oligonucleotides are the most direct means to target RNA in a selective manner and have become an established platform ...
C Frank, Bennett   +4 more
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Antisense Drug Discovery and Development

Future Medicinal Chemistry, 2011
Numerous chemically modified oligonucleotides have been developed so far and show their own unique chemical properties and pharmacodynamic/pharmacokinetic characteristics. Among all non-natural nucleotides, to the best of our knowledge, only five chemistries are currently being tested in clinical trials: phosphorothioate, 2´-O-methyl RNA, 2´-O ...
Tsuyoshi, Yamamoto   +3 more
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Antisense Drug Technology

2021
Basic Principles of Antisense Technology Stanley T. Crooke Medicinal Chemistry of Antisense Oligonucleotides P. Dan Cook Analytical Methods for Antisense Drugs Janet M. Leeds and Lendell L. Cummins A Role for Antisense Technology in the Discovery of Highly Specific and Versatile Signal Transduction Inhibitors Brett P. Monia, Erich Koller, and William A.
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Design and development of antisense drugs

Expert Opinion on Drug Discovery, 2008
With the advent of nucleic acids as therapeutic molecules, the traditional drug discovery and development process has undergone a radical change. Because nucleic acids target complementary sequences and the recognition is on the basis of Watson-Crick base pair formation, the specificity of the whole process is high.
Ravinder, Malik, Ipsita, Roy
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Antisense Oligonucleotides as Potential Drugs

2006
Antisense technology is a novel method for drug discovery and drug development. Antisense oligodeoxynucleotides (ODN) work at the genetic level by inhibiting the translation of disease-causing proteins. Proteins are the key players in the etiology of diseases.
Engels, J., Eckstein, F.
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Efficiency of Antisense Oligonucleotide Drug Discovery

Antisense and Nucleic Acid Drug Development, 2002
The costs for discovering and developing new drugs continue to escalate, with current estimates that the average cost is more than $800 million for each new drug brought to the market. Pharmaceutical companies are under enormous pressure to increase their efficiency for bringing new drugs to the market by third-party payers, shareholders, and their ...
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Comparison of antisense oligonucleotide drug delivery systems

Journal of Controlled Release, 2004
Antisense oligonucleotides (AS-ONs) are specific drugs to inhibit gene expression at the transcriptional level. They possess a poor bioavailability and can be degraded by nucleases very rapidly. Therefore, a strong need for the development of oligonucleotide drug delivery systems exists. In the present study, two commercially available liposomes (DOTAP,
Jörg, Weyermann   +2 more
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Antisense oligonucleotide technologies in drug discovery

Expert Opinion on Drug Discovery, 2006
The principle of antisense oligonucleotide (AS-OD) technologies is based on the specific inhibition of unwanted gene expression by blocking mRNA activity. It has long appeared to be an ideal strategy to leverage new genomic knowledge for drug discovery and development.
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Antisense oligonucleotides as emerging drugs

Emerging Drugs, 1998
Virtually every major human disease can be characterised by the discordant over-expression of one or more genes whose protein products contribute to the underlying pathophysiology. Antisense oligonucleotides provide a direct means with which to attenuate such discordant gene expression and epigenetically modify disease.
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