{"id":2001,"date":"2025-05-13T12:25:51","date_gmt":"2025-05-13T10:25:51","guid":{"rendered":"https:\/\/sqy-therapeutics.com\/science-2\/"},"modified":"2025-09-17T13:34:15","modified_gmt":"2025-09-17T11:34:15","slug":"science-en","status":"publish","type":"page","link":"https:\/\/sqy-therapeutics.com\/en\/science-en\/","title":{"rendered":"Science"},"content":{"rendered":"\t\t
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What is Antisense<\/h2>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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Oligonucleotide (ASO) Therapy?<\/h2>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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Antisense oligonucleotide (ASO) approach is a personalized therapeutic avenue based on single-stranded synthetic nucleic acid chains targeting and modulating gene expression through complementary base pairing.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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Such oligonucleotides are designed to identify and bind with high specificity a particular sequence on messenger RNA (mRNA) or pre-mRNA and modulate its maturation and fate.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t

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According to their mechanism of action, ASOs can be categorized into several types:<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t

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Gapmer\u2010type ASO bind to their target mRNA to form DNA:RNA hybrids that recruit RNase H for the cleavage and degradation of mRNA.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t

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siRNAs enter the cell as double strands and are incorporated into the RNA-induced silencing complex (RISC), where the siRNA gets unwound. The resulting unwound siRNA bind to its target mRNA to induce cleavage of complementary mRNA and consequently block protein translation.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t

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Splice-switching ASOs bind to pre-mRNA and modulate the splicing process to exclude specific exons allowing the production of a functional protein from a mutated gene.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t

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Antisense oligonucleotides are incredibly versatile molecules offering promising new perspectives for the treatment of genetic diseases, whether rare or more common.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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ASO therapy also represents a significant advancement in the field of personalized medicine.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t

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Based on antisense oligonucleodies, exon-skipping<\/strong> is a one of the most promising therapeutic avenue<\/strong> to correct genetic defect.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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ASOs binds with high affinity on pre-messenger RNA (pre-mRNA) prior splicing (mRNA maturation process) in a sequence-specific manner.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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By interfering with key recognition elements of the splicing machinery, AONs \u201cmask\u201d the inclusion of a faulty or misaligned exon in mature RNA. This allows for \u201cskipping\u201d of the defective exon or restoration of an operational reading frame resulting in the production of a partially deleted but still functional protein. <\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t

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Although the resulting protein is not completely normal, the newly rescued protein may be functional enough to improve the disease symptoms.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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In the case of Duchenne Muscular Dystrophy, ASOs allow the removal of
a selected exon at one edge of the mutation to generate an exon-skipped DMD mRNA able
to produce a Becker Muscular Dystrophy (BMD)-like internally truncated dystrophin
retaining crucial protein functions. Exon-skipping therapy converts a severe DMD form of the
disease into a milder BMD phenotype.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t","protected":false},"excerpt":{"rendered":"

What is Antisense Oligonucleotide (ASO) Therapy? Antisense oligonucleotide (ASO) approach is a personalized therapeutic avenue based on single-stranded synthetic nucleic acid chains targeting and modulating gene expression through complementary base pairing. Such oligonucleotides are designed to identify and bind with high specificity a particular sequence on messenger RNA (mRNA) or pre-mRNA and modulate its maturation […]<\/p>\n","protected":false},"author":1,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"footnotes":""},"class_list":["post-2001","page","type-page","status-publish","hentry"],"_links":{"self":[{"href":"https:\/\/sqy-therapeutics.com\/en\/wp-json\/wp\/v2\/pages\/2001","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/sqy-therapeutics.com\/en\/wp-json\/wp\/v2\/pages"}],"about":[{"href":"https:\/\/sqy-therapeutics.com\/en\/wp-json\/wp\/v2\/types\/page"}],"author":[{"embeddable":true,"href":"https:\/\/sqy-therapeutics.com\/en\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/sqy-therapeutics.com\/en\/wp-json\/wp\/v2\/comments?post=2001"}],"version-history":[{"count":0,"href":"https:\/\/sqy-therapeutics.com\/en\/wp-json\/wp\/v2\/pages\/2001\/revisions"}],"wp:attachment":[{"href":"https:\/\/sqy-therapeutics.com\/en\/wp-json\/wp\/v2\/media?parent=2001"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}