Could a Single Oligonucleotide Medication Treat Multiple Different Diseases?
Our relatively new field is swiftly growing, and another exciting novel therapeutic RNA modality is now gaining traction. tRNA is vital in protein synthesis and by engineering tRNA molecules...
Support for Ukrainian Scientists
The OTS joins other scientific organizations in seeking to help Ukrainian scientists. Specifically, we invite Ukrainian students, scientists, and industry professionals with interests in the field of nucleic acids and oligonucleotide therapeutics to post their contact information and CVs on the OTS website. We [...]
A New ASO Shows Promise in Treating Dravet Syndrome, a Severe Form of Epilepsy
Typically, a single copy of a gene is enough to support normal growth and development but in a small subset of genes, the loss of one copy can cause serious diseases that are often difficult to treat. One of these conditions, Dravet Syndrome, has [...]
A Potent ASO Shows Encouraging Results in A Single-Patient Pilot Study for ALS
Amyotrophic lateral sclerosis (ALS) is a progressive, neurodegenerative disease in which motor neurons die. Since the motor neurons that die control voluntary muscles, the disease causes great difficulty for those who must live with it. They will experience loss of motor control of the [...]
Does CRISPR Gene Editing Increase the Risk of Developing Cancer?
CRISPR is a useful tool in a wide array of applications, and one of the most headline-grabbing is its potential in gene editing. As with any new technology or medication, there are inherent risks that must be discovered and mitigated. This is especially true [...]
Clinical Trials Begin for Ultra-Rare Treatment of Alexander Disease
It is always exciting to observe phenomenal results in the treatment of a disease in animal studies, and even more so to see them move into clinical trials. ASOs have proven to be efficient at providing deep reductions in disease causing proteins, and a [...]
Can an ASO Reduce Disease Progression in ALS?
What if you noticed your body acting a bit “off,” doing something unusual and more than a bit worrying? Then, you go to a doctor and are eventually told that you have a debilitating neurodegenerative disease and can expect to progressively lose control of [...]
IKARIA – An Innovative Platform that Yields Long-Acting siRNA
In this year that marks the 20th Anniversary of the discovery of RNA interference as a gene-silencing mechanism in mammalian cells, Alnylam announced another remarkable development in siRNA therapeutics – a platform that could allow for administration of siRNA medications just once a year [...]
New Miniature CRISPR-Cas Systems Engineered to Function in Human Cells
CRISPR-Cas systems provide a revolutionary approach to curing disease through precise genome editing and CRISPR therapies have delivered promising early successes. However, engineering more highly efficient, compact Cas systems could result in a more versatile tool that could greatly improve and expand CRISPR-based therapeutics. [...]
President’s Pick of Presentations from the Annual OTS Meeting
By: Annemieke Aartsma-Rus, Ph.D. The second virtual annual meeting of the Oligonucleotide Therapeutics Society (OTS) took place September 26-29 on a computer or laptop near you. Despite the challenges the pandemic poses on research and development in general, the oligonucleotide therapy field has made [...]
