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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
openaire   +4 more sources

Purification of antisense oligonucleotides

2000
Chromatography is an effective tool for obtaining high-purity synthetic oligonucleotides for a variety of end uses, including antisense drug therapy. Reversed-phase and anion-exchange chromatographies are widely used techniques for this application. While selectivity of these techniques can be modified by methods such as ion-pair RP-HPLC or affinity ...
R R, Deshmukh, D L, Cole, Y S, Sanghvi
openaire   +2 more sources

Therapeutic implications of antisense oligonucleotides

International Journal of Clinical & Laboratory Research, 1992
Antisense oligonucleotides and their derivatives have been shown to be specific inhibitors of gene expression. They are considered a very promising new generation of drugs, potentially useful in most human diseases, including cancers and viral infections.
D. Lattuada   +3 more
openaire   +3 more sources

Antisense oligonucleotide therapy in an individual with KIF1A-associated neurological disorder

Nature Medicine
KIF1A-associated neurological disorder (KAND) is a neurodegenerative and often lethal ultrarare disease with a wide phenotypic spectrum associated with largely heterozygous de novo missense variants in KIF1A.
Alban Ziegler   +13 more
semanticscholar   +1 more source

History of Antisense Oligonucleotides

2003
Biological science is a rapidly flowing experimental stream, at times encountering a dam that impedes further progress. At such a pomt, a single crack may induce a major breakthrough Discovery of the double helical structure of DNA in 1953 (1) caused such an event, with flooding of new information into the area now known as molecular biology.
openaire   +2 more sources

Antisense Oligonucleotide-Conjugated Nanostructure-Targeting lncRNA MALAT1 Inhibits Cancer Metastasis.

ACS Applied Materials and Interfaces, 2018
Metastasis-associated lung adenocarcinoma transcript 1 (MALAT1), a long noncoding RNA (lncRNA) located in the cell nucleus, is a critical regulator of tumor cell migration. Antisense oligonucleotides (ASOs), which can downregulate the expression level of
Ningqiang Gong   +3 more
semanticscholar   +1 more source

Antisense oligonucleotides.

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 ...
openaire   +3 more sources

Structural modifications of antisense oligonucleotides

Il Farmaco, 2003
Antisense oligonucleotides are efficient tools for the inhibition of gene expression in a sequence specific way. Natural oligonucleotides are decomposed rapidly in biological systems, which strongly restrict their application. In contrast, artificial oligonucleotides are designed to be more stable against degradation than the target mRNA, which results
Urban, Ernst, Noe, Christian
openaire   +3 more sources

ANTISENSE OLIGONUCLEOTIDES AS THERAPEUTIC AGENTS

Pharmacological Research, 1997
The potential for modulating gene expression by the use of antisense oligonucleotides has become increasingly interesting in recent years. Antisense oligonucleotides are complementary nucleic acid fragments that hybridize to target sequences within RNA to form a DNA-RNA duplex, resulting in the block of translation of messenger RNA into the protein ...
ALAMA A   +3 more
openaire   +5 more sources

Antisense Oligonucleotide Therapy in Urology

Journal of Urology, 2002
Antisense oligonucleotides are short modified DNA or RNA molecules designed to bind selectively messenger RNA and inhibit synthesis of the encoded protein. In the last 20 years antisense technology has emerged as an exciting and promising strategy, especially for treating cancer.
I, Kausch, A, Böhle
openaire   +2 more sources

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