Chemistry, structure and function of approved oligonucleotide therapeutics

27 September 24 Articles ، Muthiah Manoharan RNAi ، ASO ، OligonucleotideTherapeutics ، SpliceSwitching ، MOE ، Phosphorothioate ، GalNAcConjugation ، DrugDelivery ، ClinicalSuccess ، درمان_الیگونوکلئوتید ، مداخله_گر_آر_ان_آ ، تحویل_دارو ، فسفوروتیوآت
Chemistry, structure and function of approved oligonucleotide therapeutics
1. Introduction: In the last 25 years, 18 oligonucleotide therapeutics have been approved for treating diseases like spinal muscular atrophy, Duchenne muscular dystrophy, and hereditary transthyretin-mediated amyloidosis. These therapies operate through different mechanisms, including antisense oligonucleotides (ASOs), splice-switching oligonucleotides (SSOs), RNA interference (RNAi), and RNA aptamers. 2. Historical Background: Oligonucleotide therapeutics trace back to the 1950s and 1960s with early discoveries in DNA and RNA synthesis. Pioneers like Todd, Khorana, …
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Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides

15 September 24 Articles ، Annemieke Aartsma-Rus GeneTherapy ، ExonSkipping ، ASO ، PersonalizedMedicine ، NOf1Therapy ، آنتی‌سنس_الیگو‌نوکلئوتید ، پریدن_اکسون ، درمان_ژنی ، پزشکی_شخصی_سازی_شده ، درمان_برای_یک_فرد
Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides
    Personal take on this article: This paper outlines consensus guidelines for the design and testing of antisense oligonucleotides (ASOs) specifically tailored to individual patients, known as N-of-1 therapies. ASOs are short strands of synthetic DNA that can modify gene expression by altering pre-mRNA splicing, offering potential treatments for various genetic diseases. The paper highlights the importance of designing ASOs that skip certain exons to restore protein function, which …
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