What are oligonucleotide-based therapies?
Oligonucleotide-based therapies are advanced novel interventions used in the management of various respiratory diseases such as asthma and Chronic Obstructive Pulmonary Disease (COPD). These agents primarily act by gene silencing or RNA interference.
How are oligonucleotides administered?
Consequently, the most promising results in addressing CNS diseases have come through direct administration of oligonucleotides, usually by intrathecal injection. When administered by this route oligonucleotides distribute broadly in the CNS and are taken up by both neurons and glial cells (68).
Are oligonucleotides gene therapy?
Antisense oligonucleotides (ASOs) are short oligonucleotides that can modify gene expression and mRNA splicing in the nervous system. The FDA has approved ASOs for treatment of ten genetic disorders, with many applications currently in the pipeline.
Where are nucleosides found?
Nucleosides can be produced from nucleotides de novo, particularly in the liver, but they are more abundantly supplied via ingestion and digestion of nucleic acids in the diet, whereby nucleotidases break down nucleotides (such as the thymidine monophosphate) into nucleosides (such as thymidine) and phosphate.
What is the role of oligonucleotide primers?
Oligonucleotides are used as probes for detecting specific sequences that are complementary to the oligonucleotides. Oligonucleotides made up of 2′-deoxyribonucleotides are the molecules used in polymerase chain reaction (PCR). These are referred to as primers and are used to massively amplify a small amount of DNA.
Where are nucleosides used?
Nucleosides are important biological molecules that function as signaling molecules and as precursors to nucleotides needed for DNA and RNA synthesis. Synthetic nucleoside analogues are used clinically to treat a range of cancers and viral infections.
What are nucleosides give examples?
Examples of nucleosides include cytidine, uridine, guanosine, inosine thymidine, and adenosine. A beta-glycosidic bond binds the 3′ position of the pentose sugar to the nitrogenous base. Nucleosides are used as anticancer and antiviral agents.
What is an oligonucleotide therapeutic?
Oligonucleotide therapeutics are drugs that manipulate gene expression and improve the disease state. Antisense oligonucleotides hybridize with a target mRNA to downregulate gene expression via an RNase H-dependent mechanism.
What is antisense oligonucleotide (AON) therapy?
Often these mistakes (mutations) result in inherited diseases. Antisense oligonucleotide (AON) therapy is a form of therapy that aims to correct the genetic defect at the RNA level. Since AONs act on the RNA level, the effect is, in contrast to gene therapy, temporary and retreatment is required.
How do oligonucleotides affect gene expression?
Oligonucleotide therapeutics in cancer Alterations in pre-mRNA splicing can have profound effects on gene expression and lead to cellular transformation. Oligonucleotide therapeutics are drugs that manipulate gene expression and improve the disease state. Antisense oligonucleotides hybridize with a target mRNA to downregulate gene expres …
Can oligonucleotides be used to manipulate protein production?
Future Perspectives The use of oligonucleotides to manipulate protein production has become a prominent therapeutic strategy for the treatment of genetic disorders and cancer.