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 ask our membership to spread ...
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 no treatment options that directly target ...
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 arms and legs which causes them ...
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 in the arena of gene-editing, as ...
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 treatment that produced significant results ...
Looking back… Looking forward
This will be my last contribution that I write as President of our Society. On January 1st, 2022, I will become ‘Immediate Past-President’. David Corey (University of Texas, Southwestern) will take over as President and Richard Geary (Ionis Pharmaceuticals) will become the Society’s next President-Elect. As we delayed this ...
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 your muscles. Your quality of life ...
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 with highly potent knockdown of ...
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. As Dr. Stanley Qi pointed out, ...
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 ...
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 ...
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 ...
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, ...
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 ...
Looking back… Looking forward
This will be my last contribution that I write as President of our Society. On January 1st, 2022, I will become ‘Immediate Past-President’. David Corey (University of Texas, Southwestern) ...
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 ...
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 ...
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 ...
