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Antisense oligonucleotide (ASO) is a single-stranded DNA/RNA (15-25 nt) designed to bind complementary mRNA target, ultimately facilitating its degradation. ASO drugs bear great therapeutic potential toward treatment of various diseases by altering RNA and/or reducing, restoring, and modifying protein expression through multiple molecular mechanisms. As of now, six ASO drugs have been approved by FDA to treat various diseases such as CMV retinitis, familial hypercholesterolemia, veno-occlusive disease, Duchenne muscular dystrophy, spinal muscular atrophy and familial amyloid polyneuropathy.
The goal of the ASO drugs is the downregulation of molecular target. The two most widely-used ASOs are double-stranded ASO and single-stranded ASO. The former uses the RISC complex to degrade RNA, while the latter silences gene expression by a variety of mechanisms, including the following:
Fig1. Single-stranded ASO mechanism of action.
Currently, ASOs drugs have been extensively chemically modified to increase stability, affinity, specificity and delivery while decreasing the potential for off-target effects. The chemical modifications of ASOs are generally classified as first generation, second generation, and third generation.
IntegrateRNA has many years of experience in RNA research, providing professional and high quality services and products for RNA basic research, drug discovery and clinical research. We provide ASO synthesis, in vitro screening and in vivo testing services. We guarantee strict quality control and deliver your RNA services/products in the most efficient time to meet your needs. If you have any question, please feel free to contact us.