What Is Antisense Therapeutics?

Antisense therapeutics is a form of treatment for genetic disorders or infections. When the genetic sequence of a particular gene is known to be causative of a particular disease, it is possible to synthesize a strand of nucleic acid (DNA, RNA or a chemical analogue) that will bind to the messenger RNA (mRNA) produced by that gene and inactivate it, effectively turning that gene "off". This is because mRNA has to be single stranded for it to be translated. Alternatively, the strand might be targeted to bind a splicing site on pre-mRNA and modify the exon content of an mRNA.[1]

This synthesized nucleic acid is termed an "anti-sense" oligonucleotide because its base sequence is complementary to the gene's messenger RNA (mRNA), which is called the "sense" sequence (so that a sense segment of mRNA " 5'-AAGGUC-3' " would be blocked by the anti-sense mRNA segment " 3'-UUCCAG-5' ").

Antisense drugs are being researched to treat cancers (including lung cancer, colorectal carcinoma, pancreatic carcinoma, malignant glioma and malignant melanoma), diabetes, ALS, Duchenne muscular dystrophy and diseases such as asthma and arthritis with an inflammatory component. Most potential therapies have not yet produced significant clinical results, though one antisense drug, fomivirsen (marketed as Vitravene), has been approved by the US Food and Drug Administration (FDA) as a treatment for cytomegalovirus retinitis.

Kinovate Life Sciences, Inc. and Nitto Denko Corporation Announce Launch of NittoPhase®HL – High Loaded Solid Support for Oligonucleotide S
NittoPhase® HL Loaded up to 400umol/g delivers Market Leading Economies to Reduce Synthesis Costs and Increase Yields

Kinovate Life Sciences, Inc. and Nitto Denko Corporation Announce Successful Development of NittoPhase™ HL High Loading Solid Support for Oligon
NittoPhase™ HL Loaded up to 400umol/g delivers Market Leading Economies to Reduce Synthesis Costs and Increase Yields

Dalton Pharma Services and ProCitius Announce a Strategic Alliance to Provide Contract Chemistry Services
Dalton Pharma Services and ProCitius Research jointly announce a strategic alliance created to provide clients with contract chemistry services of exceptional value.

Scholarship Honours York University Chemistry Professor
Peter Pekos, president and CEO of Dalton Pharma Services, has established a new scholarship to benefit chemistry students at York University in Toronto, in honour of his former mentor and business partner, Dr. Douglas Butler.

First Treatment for Muscular Dystrophy in Sight: Scientists Harness Exon-Skipping in Large Animal to Successfully Treat Duchenne Muscular Dystrophy
Researchers from Children's National Medical Center and colleagues in Tokyo publish results, video of first successful trial in dogs with Duchenne muscular dystrophy