Positive Results from Tofersen VALOR Trial and OLE Integrated Data
People diagnosed with the progressive neurodegenerative disease ALS previously had no hope of an effective treatment that could delay the onset of symptoms or extend their lifespan. However, as a result of recent advances in science and medicine, multiple oligonucleotide therapeutics are being developed ...
CRISPR Babies and the Scientist Behind Their Editing
At an international genome-editing summit in Hong Kong, MIT Technology Review reporter Antonio Regalado uncovered the existence of the CRISPR baby project. The now infamous scientist, He Jiankui, had used CRISPR-Cas9, a genetic engineering tool, to edit the CCR5 gene of three embryos with ...
CRISPR Patent Rights and Their Effect on the Industry
A decade ago, Jennifer Doudna of the University of California, Berkley and Emmanuelle Charpentier of the Max Planck Institute for Infection Biology drafted blueprints for a groundbreaking gene editing technique. The two scientists had found a way to cut and make precise changes to ...
Could Artificial Intelligence Provide the Key to Life Saving Medicines?
Drug discovery is time consuming and expensive, requiring trial and error screening. This means that many people have to wait decades, or a lifetime, to find a treatment for their disease. Others, especially those with rare diseases, may never have hope of a treatment. ...
A Novel Cocktail Drug Penetrates Heart Muscle and May Treat Nearly Half of All Patients with DMD
Duchenne muscular dystrophy (DMD) is a devastating disease that causes the body's muscle tissues to progressively waste away until all mobility is lost. Eventually, the heart muscle becomes fatty and fibrotic, typically leading to heart failure and death by age 30. There is no ...
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 no ...
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 ...
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 ...
