What are antisense oligonucleotides used for?
Small pieces of DNA or RNA that can bind to specific molecules of RNA. This blocks the ability of the RNA to make a protein or work in other ways. Antisense oligonucleotides may be used to block the production of proteins needed for cell growth.
What is antisense therapy used for?
Antisense therapy is an approach to fighting diseases using short DNA-like molecules called antisense oligonucleotides. Recently, antisense therapy has emerged as an exciting and promising strategy for the treatment of various neurodegenerative and neuromuscular disorders.
What are oligonucleotide-based therapies?
“Oligonucleotide therapeutics” is a general term for state-of-the-art, molecular-target agents that employ chemically synthesized oligonucleotides with a single-stranded deoxyribonucleic acid (DNA) or ribonucleic acid (RNA) backbone with potential specificity.
Can antisense oligonucleotides cross the blood brain barrier?
Antisense oligonucleotide therapies get delivered across the blood-brain barrier. August 13, 2021 — Researchers have demonstrated that heteroduplex oligonucleotide drugs conjugated with cholesterol efficiently cross the blood-brain barrier and can be used to treat central nervous system (CNS) diseases.
Are antisense oligonucleotides gene therapy?
Genetic therapies such as antisense oligonucleotide (ASOs) and RNA interference (RNAi) do exactly this. Through canonical Watson-Crick base pairing (figure 1A), these drugs bind individual RNAs to modulate gene expression and thus protein availability.
Are oligonucleotides biologics?
Biologic drugs include monoclonal antibodies, hormones, growth factors, enzymes, vaccines, oligonucleotides and other large molecule therapeutics.
How antisense technology is applicable for genetic disorders treatment?
Perhaps the most widely discussed application of antisense technology lies in its applications to gene therapy. In this case, a variety of vectors is used to introduce antisense-encoding genes into a large number of cells in a patient or animal to produce long-term inhibition of a protein.
Is an oligonucleotide a biologic?
Oligonucleotides are intermediate in size; they are bigger than small molecules, but smaller than biologics, such as peptides and proteins. Oligonucleotides are also highly soluble, and because they have a sugar phosphate backbone, they are polyanionic, highly negatively charged.
Why is the antisense strand important?
Antisense Antisense is the non-coding DNA strand of a gene. In a cell, antisense DNA serves as the template for producing messenger RNA (mRNA), which directs the synthesis of a protein.
How do oligonucleotide drugs work?
Single-stranded antisense oligonucleotides (ASOs) interact with proteins on the surface of cells and enter the cytoplasm by endocytosis. Subsequently, they escape the endosome and enter the nucleus, where they bind to the SMN2 pre-mRNA and block an intronic splicing suppressor element located in intron 7.
How do oligonucleotides work?
Synthetic oligonucleotides are negatively charged molecules with different chemical properties based on the technology used for their design. In order to regulate target gene expression, these compounds have to reach disease-associated tissues and cross cell membranes.
Is antisense therapy gene therapy?
Antisense gene therapy is a gene silencing technique similar to RNA interference, but uses a slightly different mechanism. The therapy is called a gene silencing technique because, instead of repairing the gene, it aims to “silence” the gene’s effect.