Neurodegenerative diseases and brain tumors wreak havoc on a person’s health and are notoriously difficult to treat. Oligonucleotide drugs could provide treatments if certain challenges could be overcome. Researchers at Tokyo Medical and Dental University, Takeda Pharmaceutical, and Ionis Pharmaceuticals may have found a solution.
Oligonucleotide therapies possess enormous potential as healing, life-changing, and cancer-curing drugs. However, there are many barriers to utilizing oligonucleotides to treat disease. Discovering or creating effective delivery methods for each target cell or tissue type solves many of these. There is a delivery method that is promising and highly effective – ligand conjugation.
Genome editing allows targeted, permanent changes to be made in the DNA. Oligonucleotides, by contrast, target gene transcripts, which have a high turnover. As such, repeated treatment is required for oligonucleotide therapeutics, ranging from weekly to every 6 months, depending on the target transcript and target tissue.
Oligonucleotide therapeutics have been making headlines this past year, which would not have been possible without lipid nanoparticles. This delivery system has been decades in the making and, fortunately for humanity, was developed in time to play a role in saving millions of lives.