Oligonucleotide Therapeutics Society c/o Event Innovations, Inc. 4377 Newport Avenue San Diego, CA 92107, USA Phone: (619) 795-9458 Fax: (619) 923-3230 … 15 Blocking of intronic splicing silencer N1 (ISS-N1) by an ASO fully corrects SMN2 exon 7 splicing and restores a high level of SMN from endogenous SMN2. [5] Introduction. However, oligonucleotides may be designed to enter hepatocytes and, in this case, their presence at the usual interaction sites can be assumed. Numerous development programs for oligonucleotide therapeutics are generating growing … Antisense oligonucleotide therapy (AOT/SOT) In 1978, Paul Zamecnik and Mary Stephenson reported the first experiments on antisense mechanisms of gene silencing using short synthetic antisense oligonucleotides to inhibit replication of a rales sarcoma virus … SOT utilizes short oligonucleotide sequences specific to you to target cancers and pathogens Specific oligonucleotide molecules are complementary to genetic sequences within cancer cells and pathogens; these molecules may bind to these genetic sequences, blocking expression of genes required for growth Splice-switching oligos (SSOs) can be employed to correct an irregular splicing event or to induce the expression of a new splicing variant, which may have a therapeutic function. Creating chemically stable short oligonucleotides was the earliest challenge in developing ASO therapies. Naturally occurring oligonucleotides are easily degraded by nucleases, an enzyme that cleaves nucleotides and is ample in every cell type. Currently, 10 AON therapies have been approved in the United States and Europe. Oligonucleotide-based therapeutics on the mode of their action are categorized as antisense oligonucleotides (ASOs), which are inhibitors of RNA activity, or modulators of protein activity (aptamers). A Short History of Oligonucleotide Synthesis By Richard Hogrefe, Ph.D.; TriLink BioTechnologies In order to offer an exhaustive report on the history of oligonucleotide synthesis , it would be necessary to examine the history of modern biotechnology, for the two are tightly interwoven. Creating Biomolecules for Biotech, Medical, Forensic and Clinical Applications. AB - Recent reports underscore the unparalleled potential of antisense-oligonucleotide (ASO)-based approaches to ameliorate various pathological conditions. Antisense oligonucleotide (AON) therapies involve short strands of modified nucleotides that target RNA in a sequence‐specific manner, inducing targeted protein knockdown or restoration. Already emerging as treatment for a growing list of neurologic conditions, including spinal muscular atrophy (SMA), Duchenne’s muscular dystrophy (DMD), and hereditary transthyretin amyloidosis (hATTR), antisense oligonucleotide (ASO) therapies are also of interest for treating neurodegenerative diseases. Oligonucleotide-directed splice switching is used to manipulate alternative pre-mRNA splicing. A recent update on potential therapeutic options for SMA treatment identified antisense-oligonucleotide (ASO)-based therapy as one of the most promising strategies. Oligonucleotides, or oligos, are short single or double stranded segments of nucleic acids that are linked together to form single chain biological polymers. 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