Antisense Oligos

Antisense oligos are short, synthetic nucleic acid sequences designed to bind complementary RNA, providing a targeted way to regulate gene expression in biological systems. After entering a cell, an oligo recognizes a specific messenger RNA through base pairing and can recruit RNase H to degrade the RNA or block splicing, translation, or other RNA-processing events. These mechanisms enable researchers to reduce, restore, or modify protein production with sequence-level precision. Antisense oligos support studies of gene function, RNA biology, disease mechanisms, and therapeutic development, including approaches for correcting abnormal splicing or silencing disease-associated transcripts.

Antisense Oligos - Related Videos

Research

JoVE Journal - Biology

Microinjection of mRNA and Morpholino Antisense Oligonucleotides in Zebrafish Embryos.

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Cited by 87 •

2009

Microinjection is a well-established and effective method for introducing foreign substances into fertilized zebrafish embryos. Here, we demonstrate a robust microinjection technique for performing mRNA overexpression, and morpholino oligonucleotide gene knockdown studies in zebrafish.

Research

JoVE Journal - Medicine
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Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy

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Cited by 25 •

2016

Exon skipping is currently a most promising therapeutic option for Duchenne muscular dystrophy (DMD). To expand the applicability for DMD patients and to optimize the stability/function of the resulting truncated dystrophin proteins, a multi-exon skipping approach using cocktail antisense oligonucleotides was developed and we demonstrated systemic dystrophin rescue in a dog model.

Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System

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Cited by 13 •

2019

Here, we describe a method for delivering drugs to the rat central nervous system by implanting a catheter into the lumbar intrathecal space of the spine. We focus on the delivery of antisense oligonucleotides, though this method is suitable for delivery of other therapeutic modalities as well.

Research

JoVE Journal - Medicine
Free Sample

Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides

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2020

Here, we present the molecular characterization of dystrophin 38 expression using Sanger sequencing, RT-PCR, and western blotting in the clinical trial.

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts

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Cited by 7 •

2018

Various antisense oligonucleotides (AONs) have been shown to induce exon inclusion (splice modulation) and rescue SMN expression for spinal muscular atrophy (SMA). Here, we describe a protocol for AON lipotransfection to induce exon inclusion in the SMN2 gene and the evaluation methods to determine the efficacy in SMA patient fibroblasts.

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