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Antisense oligonucleotide therapy for patients with Friedreich's ataxia carrying the c.165+5G>C splicing mutation. [PDF]
Mol Ther Nucleic AcidsYameogo P +6 more
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Nephron Experimental Nephrology, 1998
Antisense technology was developed to inhibit gene expression by utilizing an oligonucleotide complementary to the mRNA which encodes the target gene. There are a few possible mechanisms for the inhibitory effects of antisense oligonucleotides. Among them, degradation of mRNA by RNase H is considered to be the major mechanism of action for antisense ...
N, Kashihara, Y, Maeshima, H, Makino
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Antisense technology was developed to inhibit gene expression by utilizing an oligonucleotide complementary to the mRNA which encodes the target gene. There are a few possible mechanisms for the inhibitory effects of antisense oligonucleotides. Among them, degradation of mRNA by RNase H is considered to be the major mechanism of action for antisense ...
N, Kashihara, Y, Maeshima, H, Makino
openaire +2 more sources
Antisense-Oligonucleotide Therapy
New England Journal of Medicine, 1996Many pharmacologic advances involve creating compounds that bind and disable proteins. Such compounds include propranolol, which blocks the β-adrenergic receptor; cimetidine, which blocks the H2 receptor; calcium-channel blockers; angiotensin-converting–enzyme inhibitors; and inhibitors of the H+/K+–ATPase pump.
F K, Askari, W M, McDonnell
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Antisense Oligonucleotide Drug Design
Current Pharmaceutical Design, 2004Maneuvering 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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Verhandelingen - Koninklijke Academie voor Geneeskunde van Belgie, 1997
Among the approaches which have been followed to convert a natural antisense phosphodiester oligonucleotide into a potential therapeutic agent, conjugation chemistry seems to be one of the most attractive. Indeed, natural phosphodiester oligonucleotide have the ideal properties (sequence specific hybridization, RNaseH activation, low or no toxicity ...
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Among the approaches which have been followed to convert a natural antisense phosphodiester oligonucleotide into a potential therapeutic agent, conjugation chemistry seems to be one of the most attractive. Indeed, natural phosphodiester oligonucleotide have the ideal properties (sequence specific hybridization, RNaseH activation, low or no toxicity ...
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Pharmacokinetics of Antisense Oligonucleotides
Clinical Pharmacokinetics, 1995Antisense oligonucleotides are promising therapeutic agents for the treatment of life-threatening diseases. Intravenous injection of phosphodiester oligonucleotide analogue (P-oligonucleotide) in monkeys shows that the oligonucleotide is degraded rapidly in the plasma with a half-life of about 5 minutes.
S, Agrawal +3 more
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