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antisense oligonucleotides (AOs)

Systematic approach to developing splice modulating antisense oligonucleotides

Systematic approach to developing splice modulating antisense oligonucleotides

... switching antisense oligonucleotides (AOs) are gaining interest as therapeutics for a wide variety of inherited and acquired diseases, with the approvals of Eteplirsen and Nusinersen to treat ...

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Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models

Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models

... Dosing of mice. Intracerebroventricular (i.c.v.) infusions were performed as previously described (45). Surgeries were conducted according to Biogen Guidance for Aseptic Rodent Surgery. Animals were anesthetized by ...

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Respirable antisense oligonucleotides: a new drug class for respiratory disease

Respirable antisense oligonucleotides: a new drug class for respiratory disease

... Respirable antisense oligonucleotides (RASONs), which attenuate specific disease-associated mRNAs, represent a new class of respiratory therapeutics with considerable ...of antisense therapeutics ...

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Effect of Terminal Groups of Dendrimers in the Complexation with Antisense Oligonucleotides and Cell Uptake

Effect of Terminal Groups of Dendrimers in the Complexation with Antisense Oligonucleotides and Cell Uptake

... of antisense oligonucleotides (asODN) ...of antisense oligonucleotides as anticancer drugs could be a relatively easy task as they are the most dir- ect therapeutic method for mRNA blockade ...

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Brain pharmacology of intrathecal antisense oligonucleotides revealed through multimodal imaging

Brain pharmacology of intrathecal antisense oligonucleotides revealed through multimodal imaging

... Intrathecal (IT) delivery and pharmacology of antisense oligonucleotides (ASOs) for the CNS have been successfully developed to treat spinal muscular atrophy. However, ASO pharmacokinetic (PK) and ...

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Antisense oligonucleotides

Antisense oligonucleotides

... The genetic revolution led to the identification of many neu- rologic disease genes. The initial hope that finding the mutated protein and placing it into a known cellular pathway would lead to rapid development of ...

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Inhibition of dengue virus by novel, modified antisense oligonucleotides.

Inhibition of dengue virus by novel, modified antisense oligonucleotides.

... b-AS oligonucleotides at an intracellular concentration of 1 m M resulted in significantly less viral replication than the controls, at the lower ...for antisense inhibition (24), antisense ...

9

A Half-Century History of Applications of Antisense Oligonucleotides in Medicine, Agriculture and Forestry: We Should Continue the Journey

A Half-Century History of Applications of Antisense Oligonucleotides in Medicine, Agriculture and Forestry: We Should Continue the Journey

... Abstract: Antisense oligonucleotides (ASO), short single-stranded polymers based on DNA or RNA chemistries and synthesized in vitro, regulate gene expression by binding in a sequence-specific manner to an ...

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Role of the intercellular adhesion molecule 1(ICAM 1) in endotoxin induced pneumonia evaluated using ICAM 1 antisense oligonucleotides, anti ICAM 1 monoclonal antibodies, and ICAM 1 mutant mice

Role of the intercellular adhesion molecule 1(ICAM 1) in endotoxin induced pneumonia evaluated using ICAM 1 antisense oligonucleotides, anti ICAM 1 monoclonal antibodies, and ICAM 1 mutant mice

... and antisense oligonucleotides are inducing more changes in endothelial cells than simply inhibiting the function or the expression of the targeted adhesion molecule, although their effects are likely to be ...

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Reduction of integrin alpha 4 activity through splice modulating antisense oligonucleotides

Reduction of integrin alpha 4 activity through splice modulating antisense oligonucleotides

... of antisense oligonucleotides as therapeutics, there is an increasing interest in expanding the application of these compounds to many other ...modulating antisense oligonucleotides to treat ...

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Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing

Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing

... asthma. Antisense 2′-O-MOE oligonucleotides targeted to regions span- ning exon 9 were delivered to B cell lymphoma cells by electroporation ...of antisense oligonucleotides for downregulation ...

7

New treatments for myasthenia: a focus on antisense oligonucleotides

New treatments for myasthenia: a focus on antisense oligonucleotides

... Antisense oligonucleotides are designed to selectively hybridize with AChE m-RNA: this activates RNAses with subsequent degradation of the mRNA-antisense complex and aborted synthesis of the encoded ...

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The experimental use of antisense oligonucleotides: a guide for the perplexed

The experimental use of antisense oligonucleotides: a guide for the perplexed

... of oligonucleotides, but claim activity after examining only one, or at most two, ...one antisense oligomer was ...putative antisense oligonucleotides tested can be shown to be ...true ...

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Interleukin 6 antisense oligonucleotides inhibit the growth of human myeloma cell lines

Interleukin 6 antisense oligonucleotides inhibit the growth of human myeloma cell lines

... IL-6 antisense (and not sense) oligodeoxynucleotides; in the presence of 20 microM IL-6 antisense, an 80 and 95% inhibition of the proliferation of U 266 and RPMI 8226 cells was observed, ...

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Functionalized halloysite nanotube based carrier for intracellular delivery of antisense oligonucleotides

Functionalized halloysite nanotube based carrier for intracellular delivery of antisense oligonucleotides

... disorders. Antisense oligodeoxynu- cleotides [ASODNs] are single-strand DNA molecules complementary to regions of a target gene that specifically inhibit gene expression by hybridizing the gene ’ s mRNA ...

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Antisense oligonucleotides extend survival of prion-infected mice

Antisense oligonucleotides extend survival of prion-infected mice

... Active ASOs 1 and 2 delayed the onset of prion disease clinical signs by 82% and 99%, respec- tively, compared with saline (median 250 and 272 days after infection [dpi] , respectively, [r] ...

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Efficient inhibition of HIV 1 expression by LNA modified antisense oligonucleotides and DNAzymes targeted to functionally selected binding sites

Efficient inhibition of HIV 1 expression by LNA modified antisense oligonucleotides and DNAzymes targeted to functionally selected binding sites

... antisense to the single stranded loop of the DIS hairpin is very effective and sufficient to block dimerization. The inhibitory potential of the antisense oligos in vivo was dramatically improved upon LNA ...

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Circular Antisense Oligonucleotides Inhibit Growth of Chronic Myeloid Leukemia Cells

Circular Antisense Oligonucleotides Inhibit Growth of Chronic Myeloid Leukemia Cells

... antisense circle reduced the amount of bcr-abl protein in treated cells, we used immunoblot- ting. BV173 cells were cultured with antisense or nonsense circle for 1, 2, or 3 days[r] ...

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Development and prevention of neural tube defects in the mouse embryo

Development and prevention of neural tube defects in the mouse embryo

... boundary and such genes are thought to play a role in defining this junction.. 1987; McMahon et a l 1992) and exposure of embryos to antisense oligonucleotides for either gene durin[r] ...

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Components of neuronal calcium channels and their interaction with GTP-binding proteins

Components of neuronal calcium channels and their interaction with GTP-binding proteins

... currents. Antisense oligonucleotides have more recently provided a novel approach to investigate which subtypes o f G protein have the principal role in coupling receptor activation to inhibition o f ...

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